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RARE Daily: the official Global Genes blog

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

Drug Development

Pfizer Notifies Hemophilia Community of Patient Death in Study of Hympavzi

Rare Daily Staff A patient receiving Pfizer’s hemophilia drug Hympavzi as part of a long‑term extension trial […]

Drug Development

FDA Approves Grifols Treatment for Rare Bleeding Disorders

Rare Daily Staff The U.S. Food and Drug Administration has approved Grifols’ fibrinogen concentrate Fesilty for the […]

Drug Development

Rezolute Shares Tanks as Phase 3 Study in Congenital Hyperinsulinism Fails

Rare Daily Staff Rezolute shares fell around 90 percent after the company reported its experimental therapy ersodetug […]

Drug Development

FDA Touts Flexibility in Approving First Gene Therapy for WAS

Rare Daily Staff The U.S. Food and Drug Administration approved Fondazione Telethon ETS’ Waskyra, the first cell-based […]

Drug Development

FDA Grants Senti Bio RMAT Designation for AML Therapy

Rare Daily Staff The U.S. Food and Drug Administration has granted Senti Biosciences’ experimental cell therapy SENTI-202 […]

Drug Development

UCB Reports Positive Results from Phase 3 Study in CDKL5 Deficiency Disorder

Rare Daily Staff UCB reported that a phase 3 study of its seizure medication fenfluramine in children […]

Drug Development

Study Finds Capricor’s Cell Therapy Slows Arm Function Loss, Protects Heart

Rare Daily Staff Capricor Therapeutics said its experimental cell therapy for the rare neuromuscular condition Duchenne muscular […]

Drug Development

Belite Bio Reports Positive Topline Results from Pivotal Stargardt Trial

Rare Daily Staff Belite Bio reported positive topline results from its global Phase 3 DRAGON trial of […]

Drug Development

Child with Hunter Syndrome Doing Well After Experimental Gene Therapy

Rare Daily Staff Several months after a 3-year-old California boy was treated in the United Kingdom with […]

Finance

RARE Daily

CIRM Awards Cure Rare Disease $7.4 Million to Advance LGMD Gene Therapy

Rare Daily Staff The California Institute for Regenerative Medicine has awarded Cure Rare Disease a $7.4 million […]

RARE Daily

Denali Enters $275 Million Funding Agreement with Royalty Pharma

Rare Daily Staff Denali Therapeutics said it entered into a $275 million synthetic royalty funding agreement with […]

RARE Daily

Protego Raises $130 Million to Fund AL Amyloidosis Pivotal Study

Rare Daily Staff Protego Biopharma, which is pioneering first-in-class small-molecule therapeutics that reprogram protein folding to address […]

RARE Daily

AAVantgarde Closes $70.7 Million Financing

Rare Daily Staff Italy-based AAVantgarde Bio said it completed a $70.1 million (€61 million) series A financing […]

RARE Daily

Ovid Prices PIPE of up to $175 Million

Rare Daily Staff Ovid Therapeutics said it has entered into a securities purchase agreement for a PIPE […]

RARE Daily

Avidity Prices $600 Million Public Offering

Rare Daily Staff Avidity Biosciences said it priced an upsized $600 million public offering of 15 million […]

RARE Daily

CAMP4 Raises up to $100 Million in Private Placement to Advance SYNGAP1 Candidate

Rare Daily Staff CAMP4 Therapeutics said it has entered into definitive securities purchase agreements with institutional and […]

RARE Daily

Arnatar Therapeutics Emerges from Stealth with $52 million and an RPDD for Alagille Syndrome Therapy

Rare Daily Staff Arnatar Therapeutics, a company developing RNA-based therapies, said the U.S. Food and Drug Administration […]

RARE Daily

Citizen Health Raises $30 Million

Rare Daily Staff Healthcare technology provider Citizen Health said it raised $30 million in series A funding […]

Shionogi Expands Rare Disease Presence with Acquisition of ALS Therapy in $2.5B Deal

Rare Daily Staff Shionogi said it would acquire for $2.5 billion a newly established company that Tanabe […]

Deals

BioMarin to Acquire Amicus Therapeutics for $4.8 Billion

Rare Daily Staff BioMarin Pharmaceutical has agreed to acquire Amicus Therapeutics for $14.50 per share in an […]

Policy

HHS Adds DMD, MLD to Recommended Newborn Screening Panel

Rare Daily Staff The U.S. Department of Health and Human Services approved the addition of Duchenne muscular […]

Deals

Mirum Agrees to Acquire Bluejay Therapeutics

Rare Daily Staff Mirum Pharmaceuticals has entered into a definitive agreement to acquire Bluejay Therapeutics, a privately […]

Policy

House Passes Bill to Restore Rare Pediatric PRVs

Rare Daily Staff The U.S. House of Representatives unanimously passed the Mikaela Naylon Give Kids a Chance […]

Deals

Regeneron Enters Collaboration with Tessera for AATD Gene Editing Therapy

Rare Daily Staff Regeneron Pharmaceuticals has entered into a global collaboration with Tessera Therapeutics to develop and […]

What’s Happening

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 […]

Strategy

Global Genes Guide to Cooperating Among Patient Advocacy Organizations

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

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. […]

A Student’s Perspective: Lessons from the 2025 RARE Drug Development Symposium

During my first week as a Harvard College junior, I was delighted to be given the opportunity […]

Diagnosis

Global Genes Guide to Genetic Diagnosis

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

Living with a RARE Disease

Global Genes Guide to Securing a State Proclamation for Rare Disease Awareness

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

Financial Support

Global Genes Guide to Your Rare Rights

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

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

Turning a Rare Cancer into a Call to Action

Developing a New Approach to Cystic Fibrosis to Improve Outcomes

Finding Strength in Weakness

Developing Targeted Therapies for a Rare Autoimmune Disease

Drug Development

Pfizer Notifies Hemophilia Community of Patient Death in Study of Hympavzi

FDA Approves Grifols Treatment for Rare Bleeding Disorders

Rezolute Shares Tanks as Phase 3 Study in Congenital Hyperinsulinism Fails

FDA Touts Flexibility in Approving First Gene Therapy for WAS

FDA Grants Senti Bio RMAT Designation for AML Therapy

UCB Reports Positive Results from Phase 3 Study in CDKL5 Deficiency Disorder

Study Finds Capricor’s Cell Therapy Slows Arm Function Loss, Protects Heart

Belite Bio Reports Positive Topline Results from Pivotal Stargardt Trial

Child with Hunter Syndrome Doing Well After Experimental Gene Therapy

Finance

CIRM Awards Cure Rare Disease $7.4 Million to Advance LGMD Gene Therapy

Denali Enters $275 Million Funding Agreement with Royalty Pharma

Protego Raises $130 Million to Fund AL Amyloidosis Pivotal Study

AAVantgarde Closes $70.7 Million Financing

Ovid Prices PIPE of up to $175 Million

Avidity Prices $600 Million Public Offering

CAMP4 Raises up to $100 Million in Private Placement to Advance SYNGAP1 Candidate

Arnatar Therapeutics Emerges from Stealth with $52 million and an RPDD for Alagille Syndrome Therapy

Citizen Health Raises $30 Million

More Stories

Shionogi Expands Rare Disease Presence with Acquisition of ALS Therapy in $2.5B Deal

BioMarin to Acquire Amicus Therapeutics for $4.8 Billion

HHS Adds DMD, MLD to Recommended Newborn Screening Panel

Mirum Agrees to Acquire Bluejay Therapeutics

House Passes Bill to Restore Rare Pediatric PRVs

Regeneron Enters Collaboration with Tessera for AATD Gene Editing Therapy

What’s Happening

RARE Advocacy Exchange Session 8, Knowing Your Rare Rights

Global Genes Guide to Cooperating Among Patient Advocacy Organizations

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

A Student’s Perspective: Lessons from the 2025 RARE Drug Development Symposium

Global Genes Guide to Genetic Diagnosis

Global Genes Guide to Securing a State Proclamation for Rare Disease Awareness

Global Genes Guide to Your Rare Rights

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