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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 Grants Breakthrough Therapy Designation to Biogen’s SMA Therapy

Rare Daily Staff The U.S. Food and Drug Administration has granted Breakthrough Therapy designation to Biogen’s experimental […]

Drug Development

Zenas Reports Positive Results in IgG4‑RD Trial

Zenas Reports Positive Results in IgG4‑RD Trial to Date A drug designed to calm, rather than broadly […]

Drug Development

FDA Extends Review of Beren’s NPC Thearpy

Rare Daily Staff The U.S. Food and Drug Administration has extended its review of Beren Therapeutics’ New […]

Drug Development

FDA Grants Sanofi’s GD3 Drug Priority Review

Rare Daily Staff The U.S. Food and Drug Administration has granted priority review to Sanofi’s experimental therapy […]

Drug Development

Dyne Therapeutics submits FDA application for Duchenne therapy targeting exon 51

Dyne Therapeutics said it has submitted an application to the U.S. Food and Drug Administration seeking approval […]

Drug Development

Relay Reports Positive Results of Experimental Therapy for Rare Vascular Anomalies

Rare Daily Staff Relay Therapeutics said Tuesday that early clinical data suggest its experimental drug zovegalisib may […]

Drug Development

Sanofi’s AATD Experimental Drug Outperforms Standard Treatment

Rare Daily Staff Sanofi reported new data this week suggesting its experimental therapy for a rare genetic […]

Drug Development

BioMarin Reports Mixed Phase 3 Results for BMN 401 in Rare ENPP1 Deficiency

Rare Daily Staff BioMarin Pharmaceutical reported mixed results from its pivotal phase 3 ENERGY 3 trial of […]

Drug Development

FDA Places Clinical Hold on Aardvark PWS Studies

Rare Daily Staff The U.S. Food and Drug Administration has placed a full clinical hold on Ardvark […]

Finance

RARE Daily

Novellia Raises $18 Million to Expand RWD Platform

Rare Daily Staff Novellia said it raised $18 million in a series A financing round to expand […]

RARE Daily

Oak Hill Bio Raises $32.5 Million to Advance ASO in Angelman Syndrome

Rare Daily Staff Oak Hill Bio announced it has closed a $32.5 million series A financing to […]

RARE Daily

RTW Foundation Issues $573,000 in Grants to Address Gaps in Rare Disease Research

Rare Daily Staff RTW Foundation has awarded $573,000 in grants to eight rare disease organizations, underscoring the […]

RARE Daily

Latus Extends Series A Round to Raise Total of $97 Million

Rare Daily Staff Latus Bio said it raised $97 million in a series A financing to advance […]

RARE Daily

Cleveland Browns Owners Donate $12.5 Million to Advance Blood Cancer Research

Rare Daily Staff Oxford-Harrington Rare Disease Centre, the international drug discovery and development organization, said Cleveland Browns […]

RARE Daily

Climb Bio to Raise $110 Million in Private Placement

Rare Daily Staff Climb Bio said it has entered into a securities purchase agreement with a select […]

RARE Daily

Rocket Sells PRV for $180 Million

Rare Daily Staff Rocket Pharmaceuticals said it signed a definitive agreement to sell for $180 million the […]

RARE Daily

Immutrin Raises $87 Million to Advance ATTR-CM Therapy

Rare Daily Staff U.K.-based Immutrin raised $87 million (£65 million) in an oversubscribed series A financing to […]

RARE Daily

Prothena Receives $50 Million Milestone Payment from Novo for ATTR-CM Progress

Rare Daily Staff Prothena said it received a $50 million milestone payment from Novo Nordisk after reaching […]

BrainStorm Call for Updated FDA Approaches for Rare Disease Therapies in Journal Article

Rare Daily Staff BrainStorm Cell Therapeutics’ CEO has escalated the company’s long‑running dispute with the U.S. Food […]

Deals

Rallybio Agrees to Reverse Merger

Rare Daily Staff Rare disease drug developer Rallybio said it agreed to a reverse merger with Avenzo […]

Deals

Servier to Acquire Edgewise Muscular Dystrophy Business for up to $2.65 Billion

Servier to Acquire Edgewise Muscular Dystrophy Business for up to $2.65 Billion Rare Daily Staff Servier has […]

Diagnosis

AI Tool Accelerates Time to Rare Diagnosis

Rare Daily Staff Researchers at Baylor College of Medicine and Texas Children’s Hospital have developed an AI-powered […]

Regulatory

MHRA Seeks Comments on Rare Disease Framework

Rare Daily Staff The United Kingdom has unveiled sweeping plans to accelerate the development and approval of […]

Research

Scientists Develop New Gene Editing Approach Capable of Gene-Sized Insertions

Rare Daily Staff Scientists at UMass Chan Medical School have unveiled a new gene editing approach that […]

What’s Happening

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. […]

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 […]

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

Long Reads, Shorter Journeys

The Critical Role of Patient Community-Industry Partnership in Advancing a Gene Therapy

Capturing the Lived Experience of Rare Disease

Raising Awareness of Clinical Trials

Drug Development

FDA Grants Breakthrough Therapy Designation to Biogen’s SMA Therapy

Zenas Reports Positive Results in IgG4‑RD Trial

FDA Extends Review of Beren’s NPC Thearpy

FDA Grants Sanofi’s GD3 Drug Priority Review

Dyne Therapeutics submits FDA application for Duchenne therapy targeting exon 51

Relay Reports Positive Results of Experimental Therapy for Rare Vascular Anomalies

Sanofi’s AATD Experimental Drug Outperforms Standard Treatment

BioMarin Reports Mixed Phase 3 Results for BMN 401 in Rare ENPP1 Deficiency

FDA Places Clinical Hold on Aardvark PWS Studies

Finance

Novellia Raises $18 Million to Expand RWD Platform

Oak Hill Bio Raises $32.5 Million to Advance ASO in Angelman Syndrome

RTW Foundation Issues $573,000 in Grants to Address Gaps in Rare Disease Research

Latus Extends Series A Round to Raise Total of $97 Million

Cleveland Browns Owners Donate $12.5 Million to Advance Blood Cancer Research

Climb Bio to Raise $110 Million in Private Placement

Rocket Sells PRV for $180 Million

Immutrin Raises $87 Million to Advance ATTR-CM Therapy

Prothena Receives $50 Million Milestone Payment from Novo for ATTR-CM Progress

More Stories

BrainStorm Call for Updated FDA Approaches for Rare Disease Therapies in Journal Article

Rallybio Agrees to Reverse Merger

Servier to Acquire Edgewise Muscular Dystrophy Business for up to $2.65 Billion

AI Tool Accelerates Time to Rare Diagnosis

MHRA Seeks Comments on Rare Disease Framework

Scientists Develop New Gene Editing Approach Capable of Gene-Sized Insertions

What’s Happening

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

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

Global Genes Guide to Genetic 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