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RARE Daily: the official Global Genes blog
Browse the latest news, articles, and blog posts from Global Genes.
Featured
One Rare Mother’s Quest to Rewrite Her Son’s Future with a Gene Therapy
Amber Freed transformed the shock of her son’s diagnosis with an ultra-rare neurodevelopmental disorder into a determined […]
Read moreTransforming TSC Epilepsy with a Precision Therapy
Tuberous sclerosis complex is a rare genetic condition caused by changes in the TSC1 or TSC2 gene […]
Read moreBridging the Valley for Rare Disease Drug Development
The Oxford-Harrington Rare Disease Centre represents a transatlantic alliance created to bridge academic research and drug development […]
Read moreTurning a Rare Cancer into a Call to Action
Floyd Stewart was diagnosed with late-stage nasopharyngeal carcinoma, a rare, fast‑growing head and neck cancer that advanced […]
Read moreDrug Development
FDA Approves Sentynl’s Menkes Disease Therapy
Rare Daily Staff The U.S. Food and Drug Administration approved Sentynl Therapeutics’ Zycubo, the first treatment in […]
Read moreZenas Reports Positive Results from Phase 3 Study in IgG4-RD, But Stock Sinks
Rare Daily Staff Zenas BioPharma reported positive results from its Phase 3 trial of obexelimab, an experimental […]
Read moreCorcept’s Shares Sink as FDA Won’t Approve Treatment for Hypercortisolism
Rare Daily Staff The U.S. Food and Drug Administration has notified Corcept Therapeutics that it will not […]
Read moreUltragenyx, Moreo Shares Sink after Phase 3 Miss on Primary Endpoint
Rare Daily Staff Ultragenyx Pharmaceutical and its partner Moreo Biopharma said the phase 3 Orbit and Cosmic […]
Read morePfizer 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 […]
Read moreFDA 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 […]
Read moreRezolute 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 […]
Read moreFDA 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 […]
Read moreFDA 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 […]
Read moreFinance
Doudna’s Aurora Launches with $16 Million to Industrialize Personalized Genetic Therapies
Rare Daily Staff Aurora Therapeutics, a company seeking to transform personalized gene editing from a one-patient breakthrough […]
Read moreCIRM 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 […]
Read moreDenali 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 […]
Read moreProtego 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 […]
Read moreAAVantgarde Closes $70.7 Million Financing
Rare Daily Staff Italy-based AAVantgarde Bio said it completed a $70.1 million (€61 million) series A financing […]
Read moreOvid Prices PIPE of up to $175 Million
Rare Daily Staff Ovid Therapeutics said it has entered into a securities purchase agreement for a PIPE […]
Read moreAvidity Prices $600 Million Public Offering
Rare Daily Staff Avidity Biosciences said it priced an upsized $600 million public offering of 15 million […]
Read moreCAMP4 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 […]
Read moreArnatar 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 […]
Read moreMore Stories
n-Lorem Enters Collaboration with EspeRare to Expand Access to Individualized ASOs for Rare Genetic Diseases
Rare Daily Staff The n-Lorem Foundation and the Geneva-based EspeRare Foundation said they have entered a strategic […]
Read moreFinancial News Channel Launches CNBC Cures to Raise Rare Disease Awareness
Rare Daily Staff The financial news cable channel CNBC announced the launch of CNBC Cures, an effort to […]
Read moreArgenx Announces Leadership Transition
Rare Daily Staff Argenx, a drug developer focused on rare autoimmune diseases, said Karen Massey, current chief […]
Read moreShionogi 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 […]
Read moreBioMarin 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 […]
Read moreHHS 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 […]
Read moreWhat’s Happening
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 […]
Read moreRARE 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 […]
Read moreGlobal 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 […]
Read moreGlobal 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 […]
Read moreRARE 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 […]
Read moreRARE 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. […]
Read moreA 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 […]
Read moreGlobal Genes Guide to Genetic Diagnosis
This Global Genes Quick Guide is a resource for advocates focused on pressing topics causing challenges in […]
Read moreGlobal 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 […]
Read moreReports
Early and Often: Reimagining patient community engagement to improve clinical trials feasibility
The Global Genes Corporate Alliance has developed a new white paper that shows the transformative role of […]
Read moreContinuing Advocacy and Expanding Research Efforts: Global Genes 2023 Impact Report
To our community members: For both the rare disease community and Global Genes, 2023 marked a year […]
Read moreGlobal Genes 2023 RARE Impact Grants Report
Over the past decade, Global Genes’ RARE Impact Grant Program has provided grants to rare disease patient […]
Read moreMore Resources: Dictionary of Rare Diseases
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