RARE Daily - Global Genes

Articles

Business

January 21, 2020

Stealth Initiates Clinical Study of Second-Generation Mitochondrial Candidate in Neurodegenerative Diseases

Policy

January 20, 2020

FDA to Move to Online Submissions for Orphan Drug Designation Requests

Drug Development

January 17, 2020

Driving N-of-1 Therapies for Ultra-Rare Disease Patients

Drug Development

January 17, 2020

Creating the Means to an End

People & Organizations

January 16, 2020

Rare Leader: Daniel Lewi, Founder and CEO, Cure & Action for Tay-Sachs Foundation

Uncategorized

January 16, 2020

FDA Clears GeneTx and Ultragenyx to Begin Angelman Syndrome Trial

Business

January 16, 2020

Cyprium Receives Rare Pediatric Disease Designation for Menkes Therapy

Business

January 16, 2020

Raremark Expands Leadership Team

Business

January 14, 2020

Ionis Enters into Discovery and Development Deals with Empirico, Aro

Business

January 14, 2020

Nationwide Children’s Hospital Launches Affiliate to Manufacture Gene Therapies

Business

January 14, 2020

EUSA Pharma Enters Collaboration with BeiGene for Two Rare Disease Therapies

Business

January 13, 2020

Generation Bio Completes $100 Million Financing to Advance Re-Dosable Gene Therapy Platform

Deals

January 13, 2020

LogicBio Enters Collaboration with Takeda to Develop New Genome Editing Therapy

Business

January 13, 2020

Ultragenyx Reports Positive Topline Results from OTC Deficiency Gene Therapy Study

Business

January 10, 2020

Turning Good Thoughts into Good Actions

Awareness

January 10, 2020

Rare Leader: Emily Kramer-Golinkoff, Co-Founder, Emily’s Entourage

Business

January 10, 2020

Cutting the Time and Cost of Drug Discovery with MicroOrgans

Business

January 10, 2020

FDA Approves First Targeted Therapy to Treat Rare Mutation in Patients with GIST

Business

January 10, 2020

FDA Grants Aruvant Rare Pediatric Disease Designation for SCD Therapy

Business

January 9, 2020

Translational Medicine Accelerator and Cyclica Form Rare Disease Accelerator

Business

January 9, 2020

Catabasis and Duchenne UK Partner to Evaluate Experimental DMD Drug

Business

January 9, 2020

Miller Resigns from Abeona

Business

January 9, 2020

Regeneron Reports Positive Data in Mid-Stage Ultra-Rare Bone Disease Drug Study

Drug Development

January 8, 2020

Ionis Founder Launches Nonprofit to Produce N-of-1 Therapies

Business

January 8, 2020

VectivBio Launches with $35 Million to Develop Medicines for Serious Rare Diseases

Business

January 8, 2020

Apellis’ Pegcetacoplan Bests Soliris in Phase 3 PNH Study

Diagnosis

January 7, 2020

Senators Introduce Bill to Expand Access for Kids to WGS Through Medicaid

Business

January 7, 2020

Deep Genomics Raises $40 Million in Series B Financing

Business

January 7, 2020

Saniona Names SOBI’s Rami Levin as CEO

Business

January 7, 2020

Rhythm CEO to Step Down After FDA Filing

Diagnosis

January 6, 2020

NHS England Using DNA Testing to Accelerate Diagnosis for Critically Ill Kids

Business

January 6, 2020

FDA Grants QED Fast Track and Orphan Drug Designations for Rare Cancer Therapy

Business

January 3, 2020

Acquisition Combines Two Pipelines for Rare Skin Diseases

Pricing

January 3, 2020

NICE Rejects Akcea’s FCS Drug Waylivra as Too Costly

Uncategorized

January 2, 2020

Rare Leader: Christina SanInocencio, Executive Director, LGS Foundation

Business

January 2, 2020

Savara Wins Breakthrough Therapy Designation for Rare Lung Disease Therapy

Business

December 31, 2019

A Down Year for Drug Approvals, But Notable for Its Standouts

Business

December 31, 2019

Complete FDA Orphan Drug Approvals in 2019

Business

December 31, 2019

FDA Orphan Drug Designations in Q4 2019

Business

December 30, 2019

Fulcrum Enters Drug Discovery Collaboration with Acceleron for Undisclosed Pulmonary Disease

Drug Development

December 27, 2019

Programming Cells to Produce Neuroprotective Factors to Treat ALS

Diagnosis

December 26, 2019

Rare Leader, David Jacob, Founder and CEO of ThinkGenetic

In Rare Form

December 24, 2019

The Making of a Neuroscientist

Business

December 23, 2019

Sarepta In Blockbuster Deal with Roche for Ex-U.S. Rights to its Experimental DMD Gene Therapy

Business

December 20, 2019

Origin’s Rapid Path to Seek Approval for Treatment of Ultra-Rare Metabolic Condition

Business

December 20, 2019

Stealth BioTherapeutics PMM Therapy Fails in Late-Stage Trial

Business

December 20, 2019

Duke University Establishes Translational Research Collaboration with $130 Million from Deerfield

Business

December 20, 2019

Ultragenyx Sells Future Royalties on Crysvita for $320 Million

People & Organizations

December 19, 2019

Rare Leader: Charlene Son Rigby, President, STXBP1 Foundation

Business

December 18, 2019

Epirium Raises $85 Million to Develop Treatments for Rare Neuromuscular Diseases

Business

December 18, 2019

Global Blood Therapeutics and Syros Pharmaceuticals in Rare Blood Disorders Pact

Business

December 18, 2019

Chondrial Merges with Zafgen, Advances FA Candidate

Business

December 18, 2019

Eiger Initiates Rolling Submission Progeria Drug for FDA Approval

Drug Development

December 18, 2019

Nurturing a Culture for Translational Science

Business

December 17, 2019

Triplet Therapeutics Launches with $59 Million to Develop Treatments for Repeat Expansion Disorders

Uncategorized

December 17, 2019

Amylyx Reports Results from ALS Trial

Business

December 17, 2019

Wave Discontinues Development of Experimental DMD Drug

Uncategorized

December 17, 2019

Alnylam Reports Positive Results from Late-Stage PH1 Study of Lumasiran

Business

December 17, 2019

Proteostasis Reports Positive Study Results from Combination Therapy for CF

Business

December 16, 2019

BioMarin Reports Positive Final Results from Late-Stage Study of Achondroplasia Drug

Business

December 16, 2019

Biogen Reports It Will Discontinue Development of Drug for Progressive Supranuclear Palsy

Business

December 16, 2019

CHMP Issues Positive Opinion for Pfizer’s ATTR-CM Drug

Business

December 16, 2019

FDA Grants Mirum Rare Pediatric Disease Designation for Maralixibat in Alagille Syndrome

RARECast Podcasts

December 13, 2019

Developing a Common Language for Rare Diseases

Business

December 13, 2019

FDA Grants Accelerated Approval to First Targeted Treatment for Rare DMD Mutation

Diagnosis

December 12, 2019

Perkin Elmer Wins FDA Authorization for First Newborn Test for DMD

Awareness

December 12, 2019

Rare Leader: Justin Porcano, Co-Founder and Executive Director, Save Sight Now

In Rare Form

December 11, 2019

The Other Data Drug Developers Need to Consider

Business

December 10, 2019

Merck to Acquire ArQule for $2.7 Billion

Business

December 10, 2019

Quellis Biosciences Raises $17 Million to Develop Rare Disease Therapies

Awareness

December 10, 2019

Invitae and BioMarin Launch Genetic Testing Program for Skeletal Dysplasias

Business

December 10, 2019

Sanofi Presents Positive Results from Pivotal Trial of Sutimlimab in people with CAD

Business

December 10, 2019

ASH Meeting Offers Updates on Several Rare Disease Therapies in Development

Business

December 9, 2019

Jasper Therapeutics Complete $35 Million Financing to Expand Curative Therapies

Business

December 9, 2019

Healx and Boehringer Ingelheim Partner in Rare Neurological Diseases Pact

Innovation

December 9, 2019

Andra Stratton, Co-Counder of Lipodystrophy United, Discusses the Future of Rare Disease

Business

December 6, 2019

Limelight Bio Raises $75 Million to Develop Next-Gen Gene Therapies

Business

December 6, 2019

FDA Rejects Enzyvant’s Rare Disease Therapy over Manufacturing Issues

Drug Development

December 6, 2019

Why Payers Lag Drug Developers and Regulators in Embracing Real-World Evidence

Drug Development

December 5, 2019

Lost in Translation

People & Organizations

December 5, 2019

Rare Leader: Stacie Lindsey, Founder, CEO, Cholangiocarcinoma Foundation

Business

December 5, 2019

Regeneron Reports Positive Data with Pozelimab in Patients with a Rare Blood Disorder

Business

December 4, 2019

Censa Reports Positive Results in Phase 2 Trial in Patients with PKU

Business

December 3, 2019

Astellas Moves into Gene Therapy with $3 Billion Acquisition of Audentes

Business

December 2, 2019

Neurocrine and Xenon Partner to Develop Treatment for Rare Epilepsy

Innovation

December 2, 2019

Harsha Rajasimha, Co-founder of ORDI in USA, Discusses the Future of Rare Disease

Awareness

November 29, 2019

How a Brother’s Love Is Forging a Path for Customized Therapies for Rare Disease Patients

Awareness

November 29, 2019

Rare Leader: Katherine Wilemon, Founder and CEO, FH Foundation

Rare Disease

November 27, 2019

Brigham and Women’s Lauches Rare Liver Disease Center with $15 Million Gift

Business

November 27, 2019

La Jolla Pharmaceuticals Halts Development of Beta Thalassemia Drug as CEO Departs

Business

November 27, 2019

ChemoCentryx Reports Positive Data from Pivotal Trial of Avacopan in ANCA-Associated Vasculitis

In Rare Form

November 26, 2019

Leveraging Data

Business

November 26, 2019

FDA Approves Novel Treatment to Target Abnormality in Sickle Cell Disease

Business

November 25, 2019

Alexion’s Soliris Wins Japanese Approval to Treat NMOSD

Business

November 25, 2019

FDA Grants PTC Priority Review for SMA Therapy

Innovation

November 25, 2019

NEXT: Lara Bloom, CEO of The Ehlers Danlos Society, Discusses the Future of Rare Disease

Policy

November 22, 2019

Addressing the Barriers to Accessing Experimental Therapies

In Rare Form

November 22, 2019

Note to Payers: Get Real

Business

November 21, 2019

Healx Launches Rare Treatment Accelerator

Business

November 21, 2019

CRISPR Therapeutics Initiates $275 Million Public Offering

Business

November 21, 2019

FDA Grants Newron Rare Pediatric Disease Designation for Sarizotan in Rett Syndrome

People & Organizations

November 21, 2019

Rare Leader: Rich Horgan, Founder and President, Cure Rare Disease

Business

November 21, 2019

FDA Approves Alnylam’s RNAi Drug Givlaari for AHP Attacks

Business

November 20, 2019

Reata Raises $505 Million to Advance Rare Disease Drugs

Business

November 19, 2019

Dicerna and Novo Nordisk Partner in RNAi Therapies for Liver Diseases

Business

November 19, 2019

Gene Editing Treatment Shows Positive Results in First Two Treated Patients with Blood Disorders

Patient Tools

November 19, 2019

Backpack Health and Project Alive Launch Hunter Syndrome Study

Research

November 19, 2019

Rare Genetic Variants Linked to Sudden Cardiac Death

Business

November 19, 2019

FDA Approves Novartis Drug as First Targeted Therapy for SCD-Related Pain Crises

Policy

November 19, 2019

Australia Commits $37.5 Million to Develop Treatments for Rare Disorders

Business

November 18, 2019

Roche Beefs Up Its Fibrosis Pipeline in $1.4 Billion Deal to Acquire Promedior

Drug Development

November 18, 2019

FDA Grants Anavex Rare Pediatric Disease Designation

Drug Development

November 18, 2019

EMA Grants Orchard Accelerated Assessment of Therapy for Patients with MLD

Innovation

November 18, 2019

NEXT: Nicole Boice, Global Genes, Discusses the Future of Rare Disease

Rare Advocate

November 15, 2019

A Journey to Advocacy

Deals

November 15, 2019

Sarepta and StrideBio Collaborate to Advance Novel Gene Therapies

Diagnosis

November 15, 2019

mRNA Sequencing Ends 25-Year Diagnostic Odyssey

Drug Development

November 15, 2019

FDA Accepts New Drug Submission for Selumetinib in NF1, grants Priority Review

People & Organizations

November 14, 2019

Rare Leader: Kim Tuminello, Co-Founder, Assocation for Creatine Deficiencies

Drug Development

November 14, 2019

GSK Reports Nucala Reduces Flares in Patients with HES

Drug Development

November 14, 2019

Palvella Begins Phase 3 Portion of Pivotal Study in Pachyonychia Congenita

Deals

November 14, 2019

Centogene and Pfizer Collaborate for Rare Disease Drug Development

Awareness

November 13, 2019

Sharing Stories, Sharing Hope

Finance

November 13, 2019

Avidity Raises $100 Million to Advance Treatments for Rare Muscle Disorders

Deals

November 13, 2019

Merck Acquires Calporta for Up to $576 Million

Innovation

November 13, 2019

FDA Grants X4 Pharma Breakthrough Therapy Designation for WHIM Therapy

Drug Development

November 13, 2019

FDA Halts Solid Biosciences’ DMD Gene Therapy Trial

Drug Development

November 12, 2019

Reata Reports Positive Results from Pivotal Study of Alport Syndrome Therapy

Drug Development

November 12, 2019

Alnylam Initiates Pivotal Study of RNAi Therapeutic for PH1; Reports Positive Extension Study Results

Innovation

November 11, 2019

NEXT: Maria Picone, TREND Community, Discusses the Future of Rare Disease

Innovation

November 11, 2019

FDA Approves Celgene’s Therapy for Beta Thalassemia

Drug Development

November 11, 2019

Genentech’s Risdiplam Meets Primary Endpoint in Pivotal SMA Trial

Policy

November 10, 2019

NICE Recommends GW Pharmaceuticals’ Epidyolex for Rare Epileptic Seizures

People & Organizations

November 8, 2019

Turning Chronic Illness into a Fashion Statement

People & Organizations

November 7, 2019

Rare Leader: Torie Robinson, CEO, Epilepsy Sparks

Drug Development

November 7, 2019

Mirum Reports Positive Data from Long-Term Study in Alagille Syndrome

Finance

November 7, 2019

Centogene Raises $56 Million in IPO for Rare Disease Diagnostics

Finance

November 7, 2019

Arkuda Therapeutics Raises $44 Million to Treat Rare Form of Dementia

Drug Development

November 7, 2019

Constellation Presents Positive Early Data in Myelofibrosis Study

Research

November 6, 2019

NIH Awards New York State’s IBR $1.95 Million to Study Rare Diseases

Diagnosis

November 6, 2019

Genomenon Releases Updated Search Engine to Accelerate Rare and Genetic Disease Diagnosis

Patient Stories

November 4, 2019

Learning that Together, We Can Make a Difference

Innovation

November 4, 2019

NEXT: Luke Rosen, KIF1A.org, Discusses the Future of Rare Disease

Strategy

November 3, 2019

ARM Reports Europe Less Competitive in Attracting Advanced Therapy Clinical Trials

Rare Advocate

November 1, 2019

Connecting Young Adults with Rare and Chronic Conditions

Deals

October 31, 2019

Beam Therapeutics Teams with Prime Medicine for Prime Editing Technology

People & Organizations

October 31, 2019

Rare Leader: Stephanie Gebel, CEO, The Snow Foundation

Healthcare

October 31, 2019

Where Cutting-Edge Science Meets Horse-and-Buggy Culture

Regulatory

October 31, 2019

FDA Places Partial Hold on Trials of Zolgensma Injected into Spinal Fluid

Business

October 31, 2019

Catalyst Firdapse Fails in Congenital Myasthenic Syndromes Trial

Deals

October 30, 2019

Vertex Exercises Cystic Fibrosis Gene Editing Options from CRISPR Therapeutics

Drug Development

October 30, 2019

FDA, EMA Accept Genentech’s Application for Approval of NMOSD Therapy

Research

October 30, 2019

Study Shows Many Common Genetic Factors Determine Severity of Rare Genetic Diseases

People & Organizations

October 30, 2019

Cystic Fibrosis Foundation Launches $500 Million Path to a Cure Initiative

Rare Disease

October 29, 2019

Study Estimates More Than 300 Million with Rare Disease Worldwide, but Number Likely Higher

Innovation

October 29, 2019

NEXT: Stephen Kingsmore, Rady Children’s Institute for Genomic Medicine, Discusses the Future of Rare Disease

Research

October 29, 2019

Researchers Discover Monkey Model for Rare Blinding Disease

Business

October 28, 2019

FDA Grants Mirum Breakthrough Therapy Designation for Alagille Syndrome Drug

Business

October 25, 2019

Cabaletta Bio Raises $75 Million in IPO for T-Cell Therapies for Autoimmune Diseases

Research

October 25, 2019

MC10 and University of Vermont Form Partnership to Study Gait Abnormalities in Movement Disorders

Rare Caregiver

October 25, 2019

Helping Caregivers Care for Themselves

Drug Development

October 24, 2019

NIH Launches Global Collaboration to Tackle SCD, HIV with Gene Therapies

People & Organizations

October 24, 2019

Rare Leader: Patricia Weltin, CEO, Beyond the Diagnosis

Business

October 24, 2019

New Biotech Allievex Licenses BioMarin’s Sanfilippo ERT

Uncategorized

October 24, 2019

Orchard Says Interim Data Suggest Frozen Gene Therapy Similar to Fresh Formulation in MLD

Business

October 23, 2019

Moderna Receives FDA Fast Track Designation for Propionic Acidemia Program

Uncategorized

October 22, 2019

Rethinking the Costs of Rare Disease

Business

October 22, 2019

FDA Approves Vertex’s Triple Combo Cystic Fibrosis Treatment

Business

October 22, 2019

Protalix Reports Positive Interim Data in Late-Stage Fabry Study

People & Organizations

October 21, 2019

Gas Station Network Fuels Rare Disease Organization with $65,000 Donation

Business

October 21, 2019

ProQR, Rhythm Complete Public Offerings

Business

October 21, 2019

Alexion Receives FDA Approval for Ultomiris for aHUS

Innovation

October 20, 2019

NEXT: James Valentine of Hyman,Phelps & McNamara Discusses the Future of Rare Disease

Awareness

October 18, 2019

A Festival of Moving Pictures

Business

October 17, 2019

Stealth Says Experimental Drug Improves Cardiac Function in Barth Syndrome Patients

People & Organizations

October 17, 2019

Rare Leader: Ginger Spitzer, Past Executive Director, Foundation for Sarcoidosis Research

Business

October 16, 2019

AI Drug Developer Healx Secures $56 million to Launch Global Rare Disease Accelerator Program

Business

October 16, 2019

Editas and AskBio Team Up to Develop Novel Treatments for Neurological Diseases

Business

October 16, 2019

Ipsen Expands Its Rare Disease Portfolio with License for Blueprint Medicines’ FOP Treatment

Business

October 16, 2019

Alexion Adds to Rare Disease Pipeline with Acquisition of Achillion

Business

October 16, 2019

FDA Grants ProQR Rare Pediatric Disease Designation for Rare Eye Disease Therapy

People & Organizations

October 16, 2019

Global Genes Names Cox Scholarship Awardees

Business

October 15, 2019

With Rights Back in Hand, Reata Reports Positive Results for FA Therapy

Drug Development

October 14, 2019

The Mother of Invention Strikes Again

Innovation

October 14, 2019

NEXT: Bruce Bloom of Healx Discusses the Future of Rare Disease

Business

October 14, 2019

Roche Said Study Shows Its Therapy Is Superior to Commonly Used Treatment in Rare Autoimmune Disease

Business

October 11, 2019

Deep Genomics Boasts an AI First in Drug Discovery

Diagnosis

October 11, 2019

New Genomics Method Can Reveal the Causes of Rare Genetic Diseases

Diagnosis

October 11, 2019

Jumping Genes Found to Cause Rare Developmental Disorders in Children

Business

October 10, 2019

Alexion Pays Stealth $30 Million for Option to Co-Develop Late-Stage Program in Mitochondrial Diseases

Business

October 10, 2019

UCB to Acquire Ra Pharmaceuticals in Deal Valued at $2.1 Billion

Business

October 10, 2019

Reata Pays $330 Million to Reacquire Drug Rights from AbbVie

Rare Advocate

October 10, 2019

Rare Leader: Keisha Greaves, Founder, Girls Chronically Rock

Innovation

October 9, 2019

When Did Super Become a Bad Thing?

Business

October 9, 2019

Bluebird Bio and Novo Nordisk Team Up to Develop Genome Editing Therapies

Business

October 8, 2019

FDA Approves First Treatment to Increase Pain-Free Light Exposure in Rare Disorder

Business

October 8, 2019

Akcea Grants Pfizer Exclusive License to Antisense Therapy

Regulatory

October 8, 2019

FDA Awards 12 Grants to Fund Rare Disease Therapy Clinical Trials

Policy

October 8, 2019

FDA Provides $4.1 Million for Two Natural History Studies

Business

October 8, 2019

Avrobio Doses First Patient in Gene Therapy Trial for Cystinosis

Business

October 7, 2019

Zogenix Presents Positive Results for Experimental Treatment for TK2 Deficiency

Business

October 7, 2019

Audentes Therapeutics Reports Positive Data from XLMTM Gene Therapy Trial

Rare Advocate

October 4, 2019

How to Live with a Disability with Your Mojo Intact

Research

October 4, 2019

NIH Awards $31 Million in Funding to Bolster Rare Diseases Research Collaborations

Business

October 4, 2019

Sarepta Therapeutics Reports Positive Results for Limb-Girdle Gene Therapy

Research

October 4, 2019

Researchers Identify Cause of Rare Deadly Neurological Disease

Business

October 4, 2019

Nippon Shinyaku Files for Approval of DMD Exon-Skipping Drug

Business

October 4, 2019

Aeovian Raises $37 Million to Advance Therapy for Rare CNS Disease

Business

October 4, 2019

Goldfinch Enters Licensing Agreement with Takeda for Rare And Metabolic Kidney Diseases

People & Organizations

October 3, 2019

Rare Leader: Nathan Peck, CEO, Cure VCP Disease

Business

October 2, 2019

Biogen Reports Positive Results for Pre-symptomatic Treatment with Spinraza in SMA

Business

October 2, 2019

FDA Grants Fast Track Designation to Amryt Pharma for EB Therapy

Business

October 2, 2019

Two Drug Developers Targeting Rare Muscle Diseases Name New CEOs

Data Center

October 2, 2019

U.S. Food and Drug Administration Orphan Drug Approvals in 2019

Data Center

October 2, 2019

U.S. FDA Orphan Drug Designations Q3 2019

Business

October 1, 2019

NewLink Genetics and Lumos Merge to Focus on Rare Disease Therapies

Uncategorized

October 1, 2019

SOBI Acquires Rare Blood Disorder Biotech Dova Pharmaceuticals

Business

September 30, 2019

Ocugen and CanSino Biologics Enter Gene Therapy Strategic Partnership

Business

September 30, 2019

FDA Approves Ipsen’s Dysport to Treat Upper Limb Spasticity in Children

Business

September 25, 2019

Amryt Raises $60 Million as It Completes Acquisition of Aegerion

Business

September 25, 2019

FDA Grants Fast Track Designation to Aldeyra Therapeutics for PRV Therapy

Policy

September 24, 2019

Rare Disease Patients Get Nod in UN Declaration on Universal Health Coverage

Business

September 24, 2019

ArTara Reverse Merges with Proteon and Raises $42.5 Million

Business

September 24, 2019

FDA Grants Fast Track Designation to Annexon for Guillain-Barré Therapy

Business

September 23, 2019

Shake-up at Ionis-Owned Akcea Removes Top Execs

Business

September 23, 2019

EU Gives Marketing Nod to GW Pharmaceuticals for Drug to Treat Rare Epilepsies

Business

September 23, 2019

FDA Places Hold on Abeona Ahead of Pivotal Trial of Cell Therapy for RDEB

Business

September 22, 2019

Bluebird Bio Reports Positive Long-Term Data from CALD Gene Therapy Study

People & Organizations

September 19, 2019

Rare Leader: Marie Conley, Founder, The Conley Cushing’s Disease Foundation

Business

September 19, 2019

Strongbridge Biopharma Reports Late-Stage Results for Experimental Cushing’s Therapy

Innovation

September 18, 2019

Technology Reshaping the Rare Disease Landscape, but Non-Scientific Challenges Threaten to Impede Progress

Rare Advocate

September 18, 2019

Fighting for Other People’s Children

Business

September 18, 2019

Versantis Raises $16.1 Million to Develop Treatment for Liver Disease Including ACLF

Research

September 17, 2019

Scientists Find Cause, and Potential Therapeutic Strategy, for Krabbe Disease

Business

September 17, 2019

Acceleron Scraps FSHD Treatment After Mid-Stage Trial Disappointment

Business

September 17, 2019

FDA Approves CTD Holdings Expanded Access Treatment Program in NPC

Business

September 16, 2019

SpringWorks Raises $162 Million in IPO

Business

September 16, 2019

Akcea and Ionis Present Positive Early-Stage Data for Experimental ATTR Therapy

Rare Advocate

September 13, 2019

A Doctor’s Quest to Cure His Own Rare Disease

Business

September 13, 2019

Castle Creek Acquires Fibrocell for Gene Therapies to Treat Rare Skin Disorders

Business

September 13, 2019

Vertex Reaches Access Agreement with Scottish Government for CF Drugs

People & Organizations

September 12, 2019

Rare Leader: Janice Schwartz, Executive Director, Pachyonychia Congenita Project

Business

September 12, 2019

Pharvaris Raises $66 Million to Advance Oral Therapy for Hereditary Angioedema

Business

September 10, 2019

Trucode Launches to Advance Next-Generation Gene Editing Platform

Business

September 10, 2019

Passage Bio Licenses Experimental CMT Gene Therapy

Business

September 10, 2019

Mayo Clinic Enters Partnership with Google to Drive Digital Transformation of Healthcare

Regulatory

September 6, 2019

FDA Clarifies Accelerated Pathway for Medical Devices for Rare Disease Indications

Business

September 6, 2019

Takeda Issues Recall for Natpara

Business

September 6, 2019

Passage Bio Raises $110 Million to Develop Gene Therapies for CNS Diseases

Business

September 5, 2019

BridgeBio Creates New Subsidiary to Develop Therapy for Limb-Girdle Muscular Dystrophy

People & Organizations

September 5, 2019

Rare Leader: Akiva Zablocki, President, The Hyper IgM Foundation

Business

September 5, 2019

First Generic Orfadin Approved

Policy

September 4, 2019

USCIS Pulls Back Plan to Deport Undocumented Patients Getting Life-Saving Treatments

Business

September 4, 2019

Publisher Elsevier Cuts Deal with NIH’s NCATS for Use of Rare Disease Database

Policy

September 4, 2019

NICE Recommends Use of Gene Therapy for Rare Inherited Eye Disorder

Global Genes News

September 4, 2019

A Letter from Kimberly Haugstad

Business

September 4, 2019

UniQure’s Late-Stage Hemophilia B Gene Therapy Study Fully Enrolled

Business

September 3, 2019

Genfit Names New CEO

RARECast Podcasts

August 30, 2019

The Benefits Support Groups Offer Rare Disease Patients

Research

August 30, 2019

Researchers Discover New Biomarker for Rare Autoimmune Disease

Business

August 30, 2019

SpringWorks Gets Breakthrough Therapy Designation for Desmoid Tumor Drug

Rare Advocate

August 29, 2019

Rare Leader: Victoria Jackson, CEO, Guthy-Jackson Charitable Foundation

Business

August 29, 2019

Report of Patient Deaths Creates Questions about Vertex’s CF Drug

Business

August 29, 2019

Alexion’s Soliris Wins European Approval to Treat Rare CNS Disease

Policy

August 27, 2019

ARM Issues Statement of Principles on Genome Editing

Business

August 26, 2019

Zogenix to Acquire Modis Therapeutics for up to $400 Million

Innovation

August 26, 2019

New Gene-editing Tool Could Help Treat Genetic Disorders

Drug Development

August 23, 2019

Targeting the Epigenetic Drivers of Cancer

Research

August 23, 2019

Gene Linked to Rare Disorder also Plays a Key Role in Alzheimer’s

People & Organizations

August 22, 2019

Rare Leader: Isabel Jordan, Chair, Rare Disease Foundation

Business

August 22, 2019

Pfizer to Invest $500 Million in Gene Therapy Manufacturing Facility in North Carolina

Business

August 22, 2019

Chinook Raises $65 Million to Advance Precision Therapies for Rare Kidney Diseases

Business

August 22, 2019

Retrophin PKAN Therapy Fails in Late Stage Trial

Business

August 22, 2019

Sobi Sells Priority Review Voucher to AstraZeneca for $95 Million

Business

August 14, 2019

Renovacor Raises $11 Million to Advance Gene Therapy for Rare Cardiovascular Disease

People & Organizations

August 8, 2019

Rare Leader: Amber Freed, Founder and CEO, SLC6A1 Connect

Business

August 8, 2019

Bayer Bets on Cell Therapy with Buyout of BlueRock Therapeutics

Business

August 7, 2019

Novartis CEO Says He’s Committed to Rebuilding Trust After Manipulation of SMA Gene Therapy Data

Business

August 7, 2019

Akcea’ and Ionis’ Waylivra Meets Endpoints in Study in Patients with FPL

Business

August 7, 2019

Rhythm Reports Positive Pivotal Results from Rare Obesity Trials

Deals

August 7, 2019

Selecta Partners with AskBio to Develop AAV Gene Therapies That Can Be Re-Dosed

Drug Development

August 7, 2019

Critical Path Institute and NORD Launch Rare Disease Data Platform

Business

August 7, 2019

Glycomine Raises $33 Million

Business

August 7, 2019

Zynerba Cannabiol Shows Promise in Small Fragile X Study

Business

August 6, 2019

Allakos Reports Positive Results in Mid-Stage Study of Therapy for Rare GI Disorders

Global Genes

August 6, 2019

Global Genes Names Kimberly Haugstad as New CEO

Business

August 5, 2019

Pfizer’s Sickle Cell Treatment Fails in Late-Stage Trial

Drug Development

August 2, 2019

Seeking to Halt and Reverse Fibrotic Diseases

Business

August 2, 2019

Cystic Fibrosis Foundation Commits Up to $15 Million to Arcturus for mRNA Therapies

Business

August 2, 2019

Amicus Reports Positive Interim Clinical Data for Batten Disease Gene Therapy

Uncategorized

August 2, 2019

Ultragenyx Seeks FDA Approval for Long-Chain Fatty Acid Oxidation Disorders Therapy

In Rare Form

August 2, 2019

It’s a Low-Down Dirty Shame

People & Organizations

August 1, 2019

Rare Leader: Jennifer Wescoe, Executive Director, Wescoe Foundation for Pulmonary Fibrosis

Research

July 31, 2019

ANU Gets $6.9 Million Donation to Research Rare Autoimmune Condition

Business

July 26, 2019

Allergan and Editas Begin First Human Trial of CRISPER-Based Gene Editing Therapy

Business

July 26, 2019

Aeglea Receives FDA Breakthrough Therapy designation for Arginase 1 Deficiency ERT

People & Organizations

July 25, 2019

Rare Leader: Robyn De Leon, Executive Director, Connecting Families UCD Foundation

Legislation

July 25, 2019

Bipartisan Legislation Seeks to Save Government $100 Billion in Drug Costs

In Rare Form

July 25, 2019

Four Diseases with a Common Problem

Business

July 25, 2019

Iveric Bio Expands Gene Therapy Portfolio for Rare Retinal Diseases

In Rare Form

July 19, 2019

Give of Yourself

Business

July 17, 2019

FDA Grants Novartis Priority Review for Sickle Cell Therapy

Business

July 16, 2019

Recursion Pharmaceuticals Raises $121 Million to Advance Platform and Pipeline

Business

July 15, 2019

Recordati Acquires Rights to Novartis’ Rare Endocrine Disease Drugs

Business

July 8, 2019

Alexion Wins European Approval for Ultomiris to Treat PNH

Business

July 8, 2019

UniQure Says Hemophilia B Gene Therapy Raises FIX Activity up to 54 Percent of Normal

Business

June 28, 2019

BridgeBio Raises $349 Million in IPO to Fund Rare Disease Programs

Business

June 25, 2019

The Eye of the Beholder

Business

June 24, 2019

FDA Approves Vertex’s Cystic Fibrosis Treatment for Children Ages 6 to 11

Business

June 24, 2019

FDA Grants Alexion Priority Review for Ultomiris to Treat AHUS

Genomics

June 21, 2019

What Rare Disease Patients Should Understand about Genetics

Drug Development

June 11, 2019

FDA Grants Denali Therapeutics Rare Pediatric Disease Designation for Hunter Syndrome Therapy

Drug Development

June 7, 2019

A Gene Hunter Becomes a Drug Hunter Too

Business

June 7, 2019

Vertex Acquires Exonics Therapeutics, Expands Deal with CRISPR Therapeutics

Rare Advocate

June 6, 2019

Rare Leader: Sandra Bedrosian-Sermone, ADNP Kids Research Foundation

Business

June 5, 2019

Making a Case

Research

June 5, 2019

Newfound Autoimmune Syndrome Can Be Treated with Anti-Inflammatory Drugs

Drug Development

June 4, 2019

EU Grants Conditional Approval for Bluebird Bio’s Beta-Thalassemia Gene Therapy

Drug Development

June 4, 2019

FDA Greenlights Clinical Trial of Ra Pharmaceuticals Treatment of IMNM

Drug Development

June 4, 2019

FDA Grants SpringWorks Fast Track Designation for Experimental NF1 Therapy

RARECast Podcasts

May 31, 2019

The Psychological Toll Rare Diseases Can Have on Healthy Siblings

Rare Advocate

May 30, 2019

Rare Leader: Amy Brin, Executive Director, Child Neurology Foundation

Business

May 30, 2019

Amicus and Penn Expand Gene Therapy Collaboration

Business

May 30, 2019

Ayala Pharmaceuticals Raises $30 Million in Novartis-Led Round

Business

May 30, 2019

Inhibrx and Chiesi Enter Agreement for AATD Candidate Worth up to $162.5 Million

Business

May 29, 2019

Orchard Secures Exclusive Worldwide License Agreement for MPS-I Gene Therapy

Business

May 29, 2019

GenSight Will Seek Approval for Gene Therapy for Rare Eye Disease Strength of Sham Treatment

Business

May 29, 2019

Oxford Biomedica Secures up to $67.5 Million in Strategic Investment

Business

May 29, 2019

Viela Bio Enters NMSOD Collaboration with Hansoh Pharma Worth for up to $220 Million

Business

May 28, 2019

BioMarin’s Hemophilia A Gene Therapy Hits Prespecified Criteria for Regulatory Submissions

Business

May 24, 2019

FDA Approves Gene Therapy Zolgensma for SMA; Novartis Sets Price at $2.1 Million

Diagnosis

May 24, 2019

Blood Test Can Provide Earlier Diagnosis of Autism Spectrum Disorder

Rare Advocate

May 23, 2019

Rare Leader: Rob Long, Executive Director, Uplifting Athletes

Business

May 21, 2019

Reneo Raises $50 Million to Develop Therapies for Mitochondrial Diseases

Business

May 20, 2019

FDA Expands Use of Gattex to Include Short Bowel Syndrome

Business

May 15, 2019

Vertex and Kymera Enter Targeted Protein Degradation Collaboration

Business

May 14, 2019

Solid Biosciences Reports Serious Adverse Event in DMD Trial

Business

May 14, 2019

Celgene Receives Breakthrough Therapy Designation Kaposi Sarcoma Drug

Business

May 14, 2019

UniQure Reports Promising Interim Data from Study of Hemophilia B Gene Therapy

Research

May 9, 2019

A Roadmap for Rare Disease Groups

Rare Advocate

May 9, 2019

Rare Leader Profile: Mark Dant, Executive Director of The Ryan Foundation

Policy

May 9, 2019

Minnesota Senate Passes Legislation to Create Rare Disease Advisory Council

Business

May 8, 2019

Pfizer Pays $340 Million for Achondroplasia Therapy Developer Therachon

Business

May 8, 2019

Vielo Bio Reports Positive Pivotal Results of Treatment for Rare CNS Disorder

Business

May 8, 2019

Gilead Enters Strategic Collaboration with Goldfinch Bio on Kidney Diseases

Drug Development

May 7, 2019

FDA Approves First LEMS Drug for Children and Adds New Wrinkle to Pricing Controversy

Drug Development

May 7, 2019

GW Pharma Reports Positive Results for CBD Drug in Patients with Rare Severe Epilepsy

Drug Development

May 7, 2019

EC Approves Akcea and Ionis’ Waylivra for Rare Lipid Disorder

Business

May 7, 2019

European Commission Approves BioMarin’s PKU Drug Palynziq

Rare Advocate

April 25, 2019

Rare Leader: Amanda Moore, CEO, Angelman Syndrome Foundation

Drug Development

April 25, 2019

Global Sales of Orphan Drug Expected to Grow at Twice the Rate of Non-Orphan Drugs

Business

April 24, 2019

Fulcrum Sees Promise in Failed Heart Drug as Treatment for FSHD

Legislation

April 24, 2019

ACE Kids Act to Improve Coordination of Care for Medically Complex Kids

Diagnosis

April 23, 2019

A Case for Whole Genome Sequencing

Drug Development

April 23, 2019

Report Predicts Sharp Jump in Clinical Development Productivity for Rare Disease Therapies

Drug Development

April 22, 2019

Novartis Investigating Patient Death in SMA Gene Therapy Trial

Research

April 22, 2019

Study Points to Lithium as Potential Treatment for Limb Girdle Muscular Dystrophy

Awareness

April 19, 2019

A Young Rare Disease Advocate Discusses Life with an Undiagnosed Condition

Rare Advocate

April 18, 2019

Rare Leader: Emily Milligan, Executive Director, Barth Syndrome Foundation

Drug Development

April 18, 2019

St. Jude Gene Therapy “Cures” Babies with “Bubble Boy” Disease

Uncategorized

April 17, 2019

When a Cold Is a Serious Threat

Business

April 17, 2019

FDA Grants Ultragenyx Rare Pediatric Disease Designation for Experimental Therapy

Business

April 16, 2019

Fibrocell Teams with Castle Creek on RDEB Gene Therapy

Business

April 16, 2019

NICE Gives Akcea Nod for Tegsedi to Treat Ultra-Rare hAATR

Business

April 9, 2019

FDA Notifies Zogenix of Inadequacies in Dravet Syndrome Drug Application

Business

April 9, 2019

Cyclerion Taps Shire’s Andreas Busch as Chief Innovation Officer

Business

April 8, 2019

Alnylam and Regeneron Enter Blockbuster Collaboration

Business

April 8, 2019

Audentes Expands Platform and Pipeline with Programs for DMD and DM

Business

April 5, 2019

How a Patient Group Helped Drive Drug Development in Rett Syndrome

Uncategorized

April 4, 2019

Rare Leader: Julie Raskin, Executive Director, Congenital Hyperinsulism International

Business

April 4, 2019

ICER Calls Spinraza Too Pricey, Urges Fair Pricing of Zolgensma

Business

April 4, 2019

FDA Grants Abeona Fast Track Designation for Sanfilippo Type B Gene Therapy

Uncategorized

April 4, 2019

A Gene Hunter Swipes Right

Business

April 3, 2019

Dyne Therapeutics Raises $50 Million to Develop Therapies for Muscle Diseases

Business

April 3, 2019

EspeRare Partners with Dermelix to Develop Treatment for XLHED

Drug Development

April 2, 2019

Sangamo Provides Positive Updates on Hemophilia A and Beta Thalassemia Gene Therapy Trials

Business

April 2, 2019

AveXis Expands Gene Therapy Manufacturing Capacity with Acquisition

Business

April 1, 2019

EMA Grants Krystal Biotech PRIME Designation for DEB Gene Therapy

Business

April 1, 2019

FDA Grants AstraZenca and Merk’s NF1 Drug Breakthrough Therapy Designation

Drug Development

April 1, 2019

FDA Orphan Drug Designations Q1 2019

Rare Advocate

March 29, 2019

How Misperceptions Can Create Barriers to Care

Uncategorized

March 28, 2019

Rare Leader: Mary Anne Meskis, Executive Director, Dravet Syndrome Foundation

Business

March 28, 2019

Polyneuron Raises $22.6 Million to Advance Autoimmune Disease Therapies

Drug Development

March 28, 2019

Orchard Therapeutics Reports MLD Gene Therapy Shows Sustained Benefit

Awareness

March 28, 2019

Seeing Sickle Cell as a Blood Disease

Finance

March 27, 2019

Imara Raises $63 Million, Brings in Pfizer Rare Disease Head as CMO

Deals

March 27, 2019

ProQR and EB Research Form Drug Company

Research

March 27, 2019

NIH Gives $3.2 Million Grant to OMRF to Study Sarcoidosis

Drug Development

March 26, 2019

FDA Grants Promedior Breakthrough Therapy Designation for Idiopathic Pulmonary Fibrosis Drug

Drug Development

March 25, 2019

FDA Issues Draft Guidance on Natural History Studies for Rare Diseases

Business

March 22, 2019

Targeting Repeat Expansion Disorders with Next-Gen Antisense Drugs

Uncategorized

March 21, 2019

Rare Leader: Melissa Bryce Gamble, Executive Director, The Global Foundation for Peroxisomal Disorders

Business

March 21, 2019

FDA Approves Jazz Pharmaceuticals’ Narcolepsy Drug

Policy

March 21, 2019

Medicare Part D Spending on Specialty Drugs Climbs to $32.8B in 2015

Business

March 21, 2019

FDA Grants Enzyvant Fast Track and Rare Pediatric Disease Designations for Farber Disease Therapy

Business

March 20, 2019

Follow the Money

Rare Advocate

March 20, 2019

Cure SMA and Parent Project Muscular Dystrophy Form Collaboration to Collect RWD

Business

March 20, 2019

Alexion Teams Up with Zealand, Affibody, in Pair of Development Deals

Business

March 20, 2019

Pfizer Takes Stake in Gene Therapy Developer Vivet

Business

March 20, 2019

GW Pharmaceuticals Sells Priority Review Voucher for $105 Million

Drug Development

March 18, 2019

Gottlieb Calls on Industry to Modernize Approach to Clinical Trials

Drug Development

March 15, 2019

Getting Misfolding Proteins to Shape Up

Rare Advocate

March 14, 2019

Rare Leader: Ashley Valentine, Co-Founder, Sick Cells

Rare Disease

March 13, 2019

A High School Student Unlocks A Rare Disease Mystery

Research

March 13, 2019

NDF Awards $700,000 in Grants for GNE Myopathy Research and Programs

Policy

March 12, 2019

Report Calls on NICE to Use Greater Flexibility in Reviewing Orphan Drugs

Business

March 8, 2019

A Different Approach to Inhibiting the Complement System

Rare Advocate

March 7, 2019

Rare Leader: Haley Oyler, President, SETBP1 Society

Policy

March 7, 2019

Patient Group Calls on BIO to Disavow “Price Gouging” by LEMS Drugmaker

Business

March 6, 2019

Alnylam Reports Positive Results from Late-Stage Study of Givosiran in AHP

Awareness

March 6, 2019

FDA to Hold Public Meeting on the Impact of Rare Diseases

Awareness

March 6, 2019

How Maria Voermans Found Her Calling

Drug Development

March 4, 2019

Oxford, Harrington Discovery Institute Form Center to Advance Rare Disease Therapies

Business

March 4, 2019

Biogen to Acquire Nightstar Therapeutics for $800 Million

Business

March 2, 2019

Perlara to Wind Down Operations

Awareness

March 1, 2019

Looking Back in Time to Find Rare Disease Patients Today

Awareness

February 27, 2019

A Rare Disease Gets Rock Star Treatment

Business

February 27, 2019

Amicus Establishes Global Research and Gene Therapy Center of Excellence

Deals

February 25, 2019

Roche Agrees to Acquire Spark Therapeutics for $4.8 Billion

Business

February 25, 2019

Ipsen to Acquire Clementia Pharmaceuticals for up to $1.3 Billion

Business

February 22, 2019

Ionis CEO Stanley Crooke Discusses Success as a Platform Technology Company

Policy

February 21, 2019

Catalyst Pharmaceuticals Defends Price of LEMS Drug in Letter to Senator Bernie Sanders

Awareness

February 21, 2019

Rare Leader: Connie Lee, President and CEO, Angioma Alliance

Business

February 21, 2019

Avexis Plans $60 Million Expansion of Manufacturing Facility in North Carolina

Awareness

February 21, 2019

Teaching Doctors When to See If a Horse Has Stripes

Diagnosis

February 20, 2019

Global Commission Releases Plan for Shortening Rare Disease Diagnostic Odyssey

Business

February 19, 2019

Nestle Health Sciences Exercises Option for Codexis PKU Treatment

Business

February 15, 2019

Passage Bio Raises $115.5 Million to Develop Gene Therapies for CNS Disease

Business

February 15, 2019

Orchard Looks toward Harvesting Expanded Gene Therapy Pipeline

People & Organizations

February 15, 2019

Rare Leader: Lori Sames, CEO, Hannah’s Hope Fund

Drug Development

February 13, 2019

Study Questions the Value of Priority Review Vouchers as Incentives

Business

February 12, 2019

Neurogene Raises $68.5 to Advance Gene Therapies for Rare Neurological Diseases

Genomics

February 12, 2019

Combining Proteomics with Genomics Helps Pinpoint a Rare Disease

Innovation

February 12, 2019

Rare Disease Groups Launch Newborn Screening Study in Australia

Business

February 12, 2019

FDA Grants Clementia Rare Pediatric Disease Designation by FOP Drug

Awareness

February 8, 2019

Connecting Rare Disease Patients in India and the United States

Drug Development

February 8, 2019

Solid Biosciences Reports Disappointing Preliminary Results of DMD Gene Therapy Study

Drug Development

February 8, 2019

First Results of In Vivo Gene Editing Trial of MPS II Treatment Show Only Small Benefit

Business

February 7, 2019

FDA Approves Sanofi’s Cablivi for Rare Blood-Clotting Disorder aTTP, the First of a New Class of...

Rare Advocate

February 7, 2019

Rare Leader: Liz Marfia-Ash, President and Founder, GRIN2B Foundation

Awareness

February 6, 2019

The Little Girl They Met

Policy

February 5, 2019

Sen. Bernie Sanders Investigates $375,000 Price of LEMS Drug

Business

February 4, 2019

Alexion and Caelum Enter $550M Collaboration in AL Amyloidosis

Rare Disease

February 4, 2019

UK Grant Supports Diagnosis and Treatment of Rare Diseases

Innovation

February 4, 2019

ASCO Names Progress in Treating Rare Cancers Advance of the Year

Business

February 1, 2019

Regenerative Medicine Moves into the Spotlight

January 7, 2020

Happy New Year from Kimberly Haugstad

December 9, 2019