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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 Priority Review to BridgeBio’s Infigratinib

Rare Daily Staff The U.S. Food and Drug Administration has accepted BridgeBio Pharma’s application for oral infigratinib […]

Drug Development

Rafael Plans FDA Filing Despite NPC Drug Missing Main Goal in Phase 3 Trial

Rare Daily Staff Rafael Holdings said its experimental therapy Trappsol Cyclo for the rare, fatal lysosomal storage […]

Drug Development

UniQure’s Huntington’s Gene Therapy Slowing Decline at Four Years

Rare Daily Staff UniQure said that its experimental gene therapy AMT-130 for the rare neurodegenerative condition Huntington’s […]

Drug Development

FDA Approves First Treatment for MCT8 Deficiency

The U.S. Food and Drug Administration has approved Egetis Therapeutics’ Emcitate, a once-daily treatment for peripheral thyrotoxicosis […]

Drug Development

FDA Approves Mirum and Incyte’s FOP Drug Atebrioz

Rare Daily Staff The U.S. Food and Drug Administration has approved Atebrioz, a once-daily oral treatment from […]

Drug Development

Alexion to Present Long-Term Data of Experimental C5 Inhibitor in gMG

Rare Daily Staff Alexion, AstraZeneca Rare Disease, said new data on its experimental treatment gefurulimab for generalized […]

Drug Development

FDA Grants Priority Review to Genentech’s Enspryng for MOGAD

Rare Daily Staff The U.S. Food and Drug Administration has granted Priority Review to Genentech’s application to […]

Drug Development

FDA Approves IntraBio’s Aqneursa for Ataxia-Telangiectasia

Rare Daily Staff The U.S. Food and Drug Administration has approved IntraBio’s Aqneursa to treat ataxia in […]

Drug Development

FDA Approves Ultragenyx Gene Therapy for Sanfilippo Syndrome Type A

Rare Daily Staff The U.S. Food and Drug Administration has approved Ultragenyx Pharmaceutical’s one-time gene therapy, Fayuvi, […]

Finance

RARE Daily

X4 Secures up to $150 Million in Debt Financing as Chronic Neutropenia Trial Advances

Rare Daily Staff X4 Pharmaceuticals said it has entered into a loan agreement with K2 HealthVentures worth […]

RARE Daily

Ultragenyx Agrees to Sell Priority Review Voucher for $210 Million

Rare Daily Staff Ultragenyx Pharmaceutical has agreed to sell a rare pediatric disease priority review voucher for […]

RARE Daily

AlphaRose Secures NIH SBIR Fast Track Grant to Advance Rosiphersen for Bain Syndrome

AlphaRose Therapeutics said it has received a multi-million-dollar Small Business Innovation Research Fast Track grant from the […]

RARE Daily

ARPA-H Awards $35 Million to UNC, Emory to Build Rare-Disease AI Data Resource

Rare Daily Staff The Advanced Research Projects Agency for Health’s Rare Disease AI/ML for Precision Integrated Diagnostics […]

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 […]

Illumina Launches AI Tool to Help Researchers Find Overlooked Rare Disease Variants

Rare Daily Staff Illumina has introduced a new artificial intelligence tool designed to help researchers identify genetic […]

Deals

CSL, Alentis partner on Rare Kidney and Liver Disease Drug

Rare Daily Staff CSL will pay Alentis Therapeutics $355 million upfront and up to $1.2 billion in […]

Deals

Shionogi to Buy IntraBio to Expand Rare Disease Business in $2 Billion Deal

Rare Daily Staff Shionogi said it will acquire IntraBio for $2 billion, a move that would give […]

Policy

NICE Backs Sobi’s Tryngolza for Adults with Rare Genetic Triglyceride Disorder

Rare Daily Staff England’s health technology value watchdog has issued final draft guidance recommending Sobi’s Tryngolza (olezarsen) […]

Strategy

Travere CEO Dube to Step Down; Former Amicus CEO Campbell Named as Replacement

Rare Daily Staff Travere Therapeutics CEO Eric Dube will step down in December after nearly eight years […]

Policy

Bipartisan House Bill Seeks Permanent Rare Pediatric Disease Voucher Program

Rare Daily Staff A bipartisan group of House lawmakers has introduced legislation that would permanently authorize the […]

What’s Happening

Big News for RARE-X and Rare Disease Families: Citizen Health Partnership

Dear Global Genes Community, Today, I’m thrilled to share an exciting new chapter for RARE-X and for […]

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 […]

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

Targeting Immune Dysfunction in ALS

When the Patient Is Left to Connect the Dots

Enabling Patient Foundations to Build the Next Generation of Genetic Therapies

Using Exosomes to Tackle the Delivery Challenge of Genetic Medicines for the Brain

Drug Development

FDA Grants Priority Review to BridgeBio’s Infigratinib

Rafael Plans FDA Filing Despite NPC Drug Missing Main Goal in Phase 3 Trial

UniQure’s Huntington’s Gene Therapy Slowing Decline at Four Years

FDA Approves First Treatment for MCT8 Deficiency

FDA Approves Mirum and Incyte’s FOP Drug Atebrioz

Alexion to Present Long-Term Data of Experimental C5 Inhibitor in gMG

FDA Grants Priority Review to Genentech’s Enspryng for MOGAD

FDA Approves IntraBio’s Aqneursa for Ataxia-Telangiectasia

FDA Approves Ultragenyx Gene Therapy for Sanfilippo Syndrome Type A

Finance

X4 Secures up to $150 Million in Debt Financing as Chronic Neutropenia Trial Advances

Ultragenyx Agrees to Sell Priority Review Voucher for $210 Million

AlphaRose Secures NIH SBIR Fast Track Grant to Advance Rosiphersen for Bain Syndrome

ARPA-H Awards $35 Million to UNC, Emory to Build Rare-Disease AI Data Resource

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

More Stories

Illumina Launches AI Tool to Help Researchers Find Overlooked Rare Disease Variants

CSL, Alentis partner on Rare Kidney and Liver Disease Drug

Shionogi to Buy IntraBio to Expand Rare Disease Business in $2 Billion Deal

NICE Backs Sobi’s Tryngolza for Adults with Rare Genetic Triglyceride Disorder

Travere CEO Dube to Step Down; Former Amicus CEO Campbell Named as Replacement

Bipartisan House Bill Seeks Permanent Rare Pediatric Disease Voucher Program

What’s Happening

Big News for RARE-X and Rare Disease Families: Citizen Health Partnership

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

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