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RARE Daily: the official Global Genes blog

Browse the latest news, articles, and blog posts from Global Genes.

Drug Development

FDA Approves Priovant’s Lisraya as First Oral Therapy for Rare Autoimmune Muscle Disease

Rare Daily Staff The U.S. Food and Drug Administration approved Lisraya, a once-daily pill for adults with […]

Drug Development

FDA Expands Approval of J&J’s Imaavy to Include Rare Blood Disease

Rare Daily Staff The U.S. Food and Drug Administration has approved Johnson & Johnson’s Imaavy for adults […]

Drug Development

FDA Extends PDUFA Date for Capricor’s DMD Therapy

Rare Daily Staff The U.S. Food and Drug Administration has extended the target action date for its […]

Drug Development

FDA Places Clinical Hold on Regenexbio’s Experimental Hunter Syndrome Gene Therapy

Rare Daily Staff The U.S. Food and Drug Administration has placed a clinical hold on Regenexbio’s experimental […]

Drug Development

FDA Grants Accelerated Approval to Ultragenyx for GSDIa Gene Therapy

Rare Daily Staff The U.S. Food and Drug Administration has granted accelerated approval to Ultragenyx Pharmaceutical’s gene […]

Drug Development

FDA Approves Regeneron’s Pasatru for Rare Bone-Formation Disorder

Rare Daily Staff The U.S. Food and Drug Administration has approved Regeneron Pharmaceuticals’ Pasatru for adults […]

Drug Development

MHRA Expands Imcivree Authorization for Acquired Hypothalamic Obesity in UK

MHRA expands Imcivree authorization for acquired hypothalamic obesity in UK Rare Daily Staff Rhythm Pharmaceuticals said the […]

Drug Development

BioMarin Discontinues Development of Therapy for ENPP1 Deficiency

Rare Daily Staff BioMarin Pharmaceutical said it will discontinue development of its experimental enzyme replacement therapy for […]

Drug Development

FDA Approves Takeda’s First-in-Class Narcolepsy Type 1 Drug

Rare Daily Staff The U.S. Food and Drug Administration has approved Takeda’s Orzeyful, the first medicine designed […]

Finance

RARE Daily

Quoin Pharmaceuticals Prices Private Placement with up to $50 Million in Potential Proceeds

Rare Daily Staff Quoin Pharmaceuticals said it has entered into a private-placement financing expected to generate approximately […]

RARE Daily

PCORI Awards $13 Million for Study of Symptom Care in Two Rare Liver Diseases

The non-profit Patient-Centered Outcomes Research Institute awarded a five-year, $13 million grant to The University of North […]

RARE Daily

Epicrispr Raises $90 Million to Advance Epigenetic Therapy for FSHD

Rare Daily Staff Epicrispr Biotechnologies has closed a $90 million oversubscribed Series C financing to fund pivotal […]

RARE Daily

Rare Ventures Launches in Pittsburgh with $25 Milllion in Backing

Rare Daily Staff A new Pittsburgh-based initiative backed by up to $25 million in philanthropic funding aims […]

RARE Daily

ARPA-H Makes $27.7 Million Grant to IGI Led Effort to Deliver CRISPR-Based Therapies for IEIs

Rare Daily Staff The Advanced Research Projects Agency for Health has awarded up to $27.7 million to […]

RARE Daily

Royalty Pharma Acquires Interest in ATTR-CM Therapy for up to $425 million

Rare Daily Staff Royalty Pharma said that it has acquired a portion of Neurimmune’s royalty interest in […]

RARE Daily

Regenxbio Prices $100 Million Public Offering to Bolster Capital Position

Rare Daily Staff Regenxbio has priced an underwritten public offering expected to generate approximately $100 million in […]

RARE Daily

Mighty Therapeutics Secures Financing for Up to $150 Million

Rare Daily Staff Mighty Therapeutics, formerly known as Stealth BioTherapeutics, said it has secured up to $150 […]

RARE Daily

ARPA-H Awards up to $160 Million to Advance Personalized Curative Medicines for Rare Genetic Diseases

Rare Daily Staff The Advanced Research Projects Agency for Health has awarded up to $160 million in […]

Werewolf to Merge With Ambros, Raises $150 Million

Rare Daily Staff Werewolf Therapeutics said it will merge with privately held Ambros Therapeutics and that it […]

Deals

LEO Pharma to Buy Rights to Tanabe Drug for Rare Skin Diseases for up to $435 Million

Rare Daily Staff LEO Pharma has agreed to acquire worldwide rights to an experimental once-daily pill from […]

Deals

BioMarin to Buy Rare Bone Disease-Focused Alesta for up to $490 Million

Rare Daily Staff BioMarin Pharmaceutical said it will acquire Alesta Therapeutics in a deal worth up to […]

Deals

PTC Therapeutics to Acquire BLA-Stage Fabry Gene Therapy from Sangamo

Rare Daily Staff PTC Therapeutics has agreed to acquire ST-920, Sangamo Therapeutics’ BLA-stage gene therapy candidate for […]

Deals

Sentynl Secures Option for U.S. Rights to Mereo’s Alpha-1 Lung Disease Drug

Rare Daily Staff Sentynl Therapeutics has entered into an option and license agreement with Mereo BioPharma for […]

Deals

Jazz to Buy Actio Biosciences in $820 Million Deal Focused on Rare Childhood Epilepsy

Rare Daily Staff Jazz Pharmaceuticals said it will acquire Actio Biosciences for $820 million upfront, adding an […]

What’s Happening

Drug Development

Acting FDA commissioner pledges to prevent political interference in rare disease meeting

Global Genes was honored to be invited by the FDA to participate in a closed roundtable discussion […]

Drug Development

CURE SYNGAP1 announces $111,960 investment in RARE-X partnership to advance ProMMIS patient-reported outcome measure data collection – PR46

CURE SYNGAP1 is proud to announce a strategic investment of $111,960 in RARE-X to accelerate the collection of high-quality […]

Savara’s Early Access Program

Savara’s Early Access Program (EAP) for molgramostim inhalation solution (molgramostim) in Autoimmune Pulmonary Alveolar Proteinosis (Autoimmune […]

Publication Announcement — RARE-X: Advancing Rare Disease Research Through Patient-Driven Data

Global Genes is excited to announce “RARE-X: A patient-driven approach for collecting symptom and patient-reported outcome data […]

Accelerating Rare Disease Research Through Collaboration: Global Genes and Notre Dame Launch the Research Acceleration Program

Global Genes and University of Notre Dame have partnered to launch the Research Acceleration Program, a collaborative […]

Equity

RARE Advocacy Exchange Session 8, Knowing Your Rare Rights

This session recording is from Oct. 16, 2025 Rare Advocacy Exchange Session 8: Knowing the Rights for […]

Living with a RARE Disease

Global Genes Guide to Starting a Nonprofit Patient Advocacy Organization

This Global Genes Quick Guide is a resource for advocates focused on pressing topics causing challenges in […]

Diagnosis

RARE Advocacy Exchange Session 7, Getting a Precise Genetic Diagnosis

This session recording is from Sept. 18, 2025 Rare Advocacy Exchange Session 7: Getting A Precise Diagnosis […]

Living with a RARE Disease

RARE Advocacy Exchange Session 6 : Grieving a RARE Diagnosis

Rare disease grief differs from the grief we commonly associate with losing a loved one to death. […]

Reports

Diagnosis

More Resources: Dictionary of Rare Diseases

Search for a specific disease in our extensive list of rare diseases for disease-specific information, news, and resources.

Search Our Full Disease Dictionary

RARE Daily: the official Global Genes blog

Featured

Stopping Guillain-Barré Syndrome at Its Source

From Repeated Surgery to a Precision Patch

Curing Sickle Cell Before Life Begins

Spotting Neuromuscular Disease Red Flags

Drug Development

FDA Approves Priovant’s Lisraya as First Oral Therapy for Rare Autoimmune Muscle Disease

FDA Expands Approval of J&J’s Imaavy to Include Rare Blood Disease

FDA Extends PDUFA Date for Capricor’s DMD Therapy

FDA Places Clinical Hold on Regenexbio’s Experimental Hunter Syndrome Gene Therapy

FDA Grants Accelerated Approval to Ultragenyx for GSDIa Gene Therapy

FDA Approves Regeneron’s Pasatru for Rare Bone-Formation Disorder

MHRA Expands Imcivree Authorization for Acquired Hypothalamic Obesity in UK

BioMarin Discontinues Development of Therapy for ENPP1 Deficiency

FDA Approves Takeda’s First-in-Class Narcolepsy Type 1 Drug

Finance

Quoin Pharmaceuticals Prices Private Placement with up to $50 Million in Potential Proceeds

PCORI Awards $13 Million for Study of Symptom Care in Two Rare Liver Diseases

Epicrispr Raises $90 Million to Advance Epigenetic Therapy for FSHD

Rare Ventures Launches in Pittsburgh with $25 Milllion in Backing

ARPA-H Makes $27.7 Million Grant to IGI Led Effort to Deliver CRISPR-Based Therapies for IEIs

Royalty Pharma Acquires Interest in ATTR-CM Therapy for up to $425 million

Regenxbio Prices $100 Million Public Offering to Bolster Capital Position

Mighty Therapeutics Secures Financing for Up to $150 Million

ARPA-H Awards up to $160 Million to Advance Personalized Curative Medicines for Rare Genetic Diseases

More Stories

Werewolf to Merge With Ambros, Raises $150 Million

LEO Pharma to Buy Rights to Tanabe Drug for Rare Skin Diseases for up to $435 Million

BioMarin to Buy Rare Bone Disease-Focused Alesta for up to $490 Million

PTC Therapeutics to Acquire BLA-Stage Fabry Gene Therapy from Sangamo

Sentynl Secures Option for U.S. Rights to Mereo’s Alpha-1 Lung Disease Drug

Jazz to Buy Actio Biosciences in $820 Million Deal Focused on Rare Childhood Epilepsy

What’s Happening

Acting FDA commissioner pledges to prevent political interference in rare disease meeting

CURE SYNGAP1 announces $111,960 investment in RARE-X partnership to advance ProMMIS patient-reported outcome measure data collection – PR46

Savara’s Early Access Program

Publication Announcement — RARE-X: Advancing Rare Disease Research Through Patient-Driven Data

Accelerating Rare Disease Research Through Collaboration: Global Genes and Notre Dame Launch the Research Acceleration Program

RARE Advocacy Exchange Session 8, Knowing Your Rare Rights

Global Genes Guide to Starting a Nonprofit Patient Advocacy Organization

RARE Advocacy Exchange Session 7, Getting a Precise Genetic Diagnosis

RARE Advocacy Exchange Session 6 : Grieving a RARE Diagnosis

Reports

Early and Often: Reimagining patient community engagement to improve clinical trials feasibility

Continuing Advocacy and Expanding Research Efforts: Global Genes 2023 Impact Report

Global Genes 2023 RARE Impact Grants Report

More Resources: Dictionary of Rare Diseases