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RARE Daily: the official Global Genes blog
Browse the latest news, articles, and blog posts from Global Genes.
Featured
From Bloodletting to Breakthroughs in PV
Polycythemia vera is a chronic blood cancer in which bone marrow stem cells acquire mutations that drive […]
Read moreWhen Endpoints Miss the Point
Families of children with the ultra-rare mitochondrial disorder pyruvate dehydrogenase complex deficiency, or PDCD, are fighting to […]
Read moreReopening the Developmental Window in Rett Syndrome with a Gene Therapy
Rett syndrome is a rare neurodevelopmental disorder that disrupts a child’s ability to purposely use their hands, […]
Read moreA Data Strategy to Capitalize on a Multi‑Trillion Dollar Opportunity
A Data Strategy to Capitalize on a Multi‑Trillion Dollar Opportunity Rare diseases are often thought about in […]
Read moreDrug Development
Rhythm Drug Fails in Phase 3 Study in Genetic Obesity
Rare Daily Staff Rhythm Pharmaceuticals said that its late-stage study of its drug setmelanotide for rare genetic […]
Read moreUltragenyx Announces Positive 36-Week Data for OTC Gene Therapy
Rare Daily Staff Ultragenyx Pharmaceutical Inc. is reporting encouraging late-stage trial results for its experimental gene therapy […]
Read moreVertex Drug Shows Promise in Rare Kidney Disease IgA Nephropathy
Rare Daily Staff An experimental therapy from Vertex Pharmaceuticals significantly reduced key markers of kidney damage in […]
Read moreFDA Wants Additional Study from UniQure for Huntington’s Disease Gene Therapy
Rare Daily Staff The U.S. Food and Drug Administration advised gene therapy developer UniQure to run an […]
Read moreFDA Lifts Hold on Intellia’s Experimental Gene-Editing Therapy for ATTR-CM
Rare Daily Staff The U.S. Food and Drug Administration has removed a clinical hold on Intellia Therapeutics’ […]
Read moreFDA Grants Ascendis Accelerated Approval for Achondroplasia Drug
Rare Daily Staff The U.S. Food and Drug Administration has granted accelerated approval to Ascendis Pharma’s Yuviwel, […]
Read moreFDA Expands Use of BioMarin PKU Drug
Rare Daily Staff The U.S. Food and Drug Administration has granted expanded approval to BioMarin Pharmaceutical for […]
Read moreFDA Grants Tessera Fast Track, Orphan Drug Designations for AATD Gene Writing Therapy
Rare Daily Staff The U.S. Food and Drug Administration granted Tessera Therapeutics Fast Track and Orphan Drug […]
Read moreCervoMed’s Experimental Drug Selected for UK Platform Trial
Rare Daily Staff CervoMed said its lead experimental candidate, neflamapimod, has been selected for inclusion in the […]
Read moreFinance
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 […]
Read moreAtavistik Bio Adds $40 Million to Series B to Advance HHT and MPN Programs
Rare Daily Staff Atavistik Bio said it raised an additional $40 million in an extension of its […]
Read moreAntiverse Raises $9.3 Million, Enters Research Agreement with Cystic Fibrosis Foundation
Rare Daily Staff Antiverse, a biotech using artificial intelligence to design antibodies for traditionally “undruggable” targets, has […]
Read moreARPA-H to Provide Every Cure up to $76 Million for AI-Drive Drug Repurposing
Rare Daily Staff The Advanced Research Projects Agency for Health (ARPA-H) will provide up to $76 million […]
Read moreCyprium to Sell PRV for $205 Million
Rare Daily Staff Cyprium Therapeutics, a majority-owned subsidiary of Fortress Biotech, agreed to sell its Rare Pediatric […]
Read moreCIRM Approves $100 Million Plan to Accelerate Genetic Therapies for Rare Diseases
Rare Daily Staff The California Institute for Regenerative Medicine has approved a new funding program to accelerate […]
Read moreOcugen Closes $22.5 Million Offering
Rare Daily Staff Ocugen, which is developing gene therapies for blinding diseases, said it raised $22.5 million […]
Read moreMendra Launches with $82 Million to Focus on Unmet Needs in Rare Disease
Rare Daily Staff Mendra, a biopharmaceutical company seeking to advance rare disease therapies utilizing artificial intelligence, announced […]
Read moreDoudna’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 moreMore Stories
Sentynl Therapeutics to License Experimental Progeria Drug
Rare Daily Staff Sentynl Therapeutics has entered into an agreement to license an experimental drug for Hutchinson-Gilford […]
Read moreNatera Launches New WGS Test
Natera said it has launched a new genomic testing platform designed to help diagnose rare diseases faster. […]
Read morePrasad to Step Down at FDA’s CBER
Rare Daily Staff Vinay Prasad, the controversial head of the U.S. Food and Drug Administration’s Center for […]
Read moreAlfasigma to License GSK Drug for Rare Liver Disease
Rare Daily Staff Drug giant GSK has agreed to license its experimental drug linerixibat to Italian pharmaceutical […]
Read moreAvidity Spinout Launches with $270 Million, Focus on Rare Cardiomyopathies
Rare Daily Staff Atrium Therapeutics, a spinout from Avidity Biosciences, has launched as a newly independent, publicly […]
Read moreFDA Unveils Framework to Speed Up Personalized Therapies for Ultra-Rare Diseases
Rare Daily Staff The U.S. Food and Drug Administration on Monday released draft guidance outlining a new regulatory framework […]
Read moreWhat’s Happening
Savara’s Early Access Program
Savara’s Early Access Program (EAP) for molgramostim inhalation solution (molgramostim) in Autoimmune Pulmonary Alveolar Proteinosis (Autoimmune […]
Read morePublication 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 […]
Read moreAccelerating 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 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 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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