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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 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 […]

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, […]

Drug Development

Whitepaper Finds Genetic Testing in Rare Disease Studies Increasingly Used to Determine Eligibility

Rare Daily Staff A new analysis of actively recruiting clinical trials suggests that genetic testing and other […]

Drug Development

Vera Reports Positive Two-Year IgAN Data; Plans to Seek Full Approval for Trutakna

Rare Daily Staff Vera Therapeutics said its drug Trutakna slowed the loss of kidney function and reduced […]

Drug Development

FDA Launches Pilot to Help Speed First Human Studies

Rare Daily Staff The U.S. Food and Drug Administration has opened applications for a new pilot program […]

Drug Development

FDA Approves Scholar Rock’s SMA Therapy to Improve Muscle Function

Rare Daily Staff The U.S. Food and Drug Administration has approved Scholar Rock’s Isembyld for adults and […]

Drug Development

BioMarin Reports Positive Data of Voxzogo in Kids with Hypochondroplasia

Rare Daily Staff BioMarin Pharmaceutical said Voxzogo increased growth rates and improved several measures of skeletal growth […]

Drug Development

FDA Grants Fast Track Designation to Sobi’s VEXAS Therapy

Rare Daily Staff The U.S. Food and Drug Administration has granted Fast Track designation to Sobi’s pacritinib […]

Drug Development

FDA Grants Priority Review to Intellia’s One-Time CRISPR Treatment for HAE

Rare Daily Staff The U.S. Food and Drug Administration has granted Intellia Therapeutics Priority Review for lonvoguran […]

Finance

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 […]

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 […]

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 […]

Diagnosis

GeneDx, Beren Launch Sponsored Genome Testing Program for Niemann-Pick Type C

Rare Daily Staff GeneDx and Beren Therapeutics have launched a sponsored genetic testing program intended to help […]

Deals

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 […]

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

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

Building a Roadmap to Treat a Rare Neurodevelopmental Condition

The Long Road from the NICU to New Therapies

Stopping Guillain-Barré Syndrome at Its Source

Drug Development

FDA Approves IntraBio’s Aqneursa for Ataxia-Telangiectasia

FDA Approves Ultragenyx Gene Therapy for Sanfilippo Syndrome Type A

Whitepaper Finds Genetic Testing in Rare Disease Studies Increasingly Used to Determine Eligibility

Vera Reports Positive Two-Year IgAN Data; Plans to Seek Full Approval for Trutakna

FDA Launches Pilot to Help Speed First Human Studies

FDA Approves Scholar Rock’s SMA Therapy to Improve Muscle Function

BioMarin Reports Positive Data of Voxzogo in Kids with Hypochondroplasia

FDA Grants Fast Track Designation to Sobi’s VEXAS Therapy

FDA Grants Priority Review to Intellia’s One-Time CRISPR Treatment for HAE

Finance

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

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

More Stories

Bipartisan House Bill Seeks Permanent Rare Pediatric Disease Voucher Program

GeneDx, Beren Launch Sponsored Genome Testing Program for Niemann-Pick Type C

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

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