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RARE Daily: the official Global Genes blog
Browse the latest news, articles, and blog posts from Global Genes.
Featured
Why a Topical Cannabidiol Gel May Help Treat the Behavioral Symptoms of Fragile X
Fragile X syndrome is a rare, genetic, developmental disorder that is the leading known cause of both […]
Read moreAn Ultra-Rare Disease Drug Developer Tries to Navigate Regulatory Uncertainty
When Stealth Biotherapeutics sought approval for its experimental therapy to treat the ultra-rare and life-threatening condition Barth […]
Read moreA Down Year for Drug Approvals, But Notable for Its Standouts
Editor’s note: This version corrects an earlier number on orphan drugs approvals in 2019 On paper, it […]
Read moreFDA Won’t Change Approach to Orphan Exclusivity after Court Ruling
Rare Daily Staff The U.S. Food and Drug Administration published a notification in the Federal Register that […]
Read moreLatest
Genentech’s C5 Inhibitor Meets Co-Primary Endpoints in Phase 3 Study in PNH
Rare Daily Staff Roche’s Genentech said its C5 complement inhibitor crovalimab met its co-primary endpoints in a […]
Read moreJanssen Reports Positive Phase 2 Results for Nipocalimab in Pregnant Women at High Risk for Severe Hemolytic Disease of the Fetus and Newborn
Rare Daily Staff The Janssen Pharmaceutical Companies of Johnson & Johnson reported positive topline results from the […]
Read moreFDA Grants Fast Track Designation to Endogena’s EA-2353 for the Treatment of Retinitis Pigmentosa
Rare Daily Staff The U.S. Food and Drug Administration granted Fast Track designation to Endogena Therapeutics’ EA-2353 […]
Read moreFDA Approves Takeda’s Takhzyro to Prevent Hereditary Angioedema Attacks in Children 2 Years of Age and Older
Rare Daily Staff The U.S. Food and Drug Administration has approved Takeda’s supplemental Biologics License Application for […]
Read moreUse of AI Platform Improves Diagnostic Rate of Rare Diseases in Japan
Rare Daily Staff The National Center for Child Health and Development in Japan and the U.S.-based OmniTier […]
Read moreCIRM Awards $1.5 Million to Rubedo Life Sciences for IPF Program
Rare Daily Staff The California Institute for Regenerative Medicine awarded Rubedo Life Sciences a $1.5 million grant […]
Read moreCerta Reports Positive Phase 2 Results in Scleroderma
Rare Daily Staff Certa Therapeutics reported positive results from a phase 2 study of FT011, its experimental […]
Read moreVyant Bio Begins Cash Preservation Plan, Potentially Winding Down Operations
Rare Daily Staff Vyant Bio, a company focused on neurodevelopmental and neurodegenerative disorders, said its board of […]
Read moreFDA Places Hold on 4D Molecular Gene Therapy for Fabry Disease
Rare Daily Staff 4D Molecular said the U.S. Food and Drug Administration placed a clinical hold on […]
Read moreDrug Development
FDA Grants Fast Track Designation to Reneo’s Mavodelpar in a Genotype of LC-FAOD
Rare Daily Staff The U.S. Food and Drug Administration has granted Fast Track designation to Reneo Pharmaceuticals’ […]
Read moreFDA Grants Breakthrough Therapy Designation to Abbisko’s Pimicotinib
Rare Daily Staff The U.S. Food and Drug Administration granted Abbisko Therapeutics Breakthrough Therapy designation for pimicotinib, […]
Read moreFDA Grants Fast Track Designation to Syros’ Tamibarotene for Treatment Group of Rare Cancers
Rare Daily Staff The U.S. Food and Drug Administration granted Fast Track designation to Syros Pharmaceuticals’ tamibarotene […]
Read moreMagenta Pauses Dose Escalation Trial of Conditioning Agent Following Patient Death
Rare Daily Staff Magenta Therapeutics said it voluntarily paused a dose escalation trial of its conditioning agent […]
Read moreCapricor Reports Positive 18-Month Results from Ongoing DMD Study
Rare Daily Staff Capricor Therapeutics said 18-month results from its ongoing HOPE-2 open label extension study in […]
Read moreCadrenal Granted FDA Fast Track Designation for Tecarfarin for Prevention of Blood Clots
Rare Daily Staff The U.S. Food and Drug Administration granted a Fast Track designation to Cadrenal Therapeutics’ […]
Read morePliant Reports Positive Data from Phase 2a Trial of Bexotegrast in Patients with IPF
Rare Daily Staff Pliant Therapeutics reported 12-week interim data from a phase 2a clinical trial of bexotegrast […]
Read moreFDA Grants Blueprint Medicines Priority Review for Ayvakit in Expanded Indication
Rare Daily Staff The U.S. Food and Drug Administration granted priority review to Blueprint Medicines’ Ayvakit for […]
Read moreFDA Lifts Hold on Astellas’ Pompe Gene Therapy
Rare Daily Staff
The U.S. Food and Drug Administration lifted the clinical hold it placed on Astellas Pharma’s FORTIS phase […]
Read moreFinance
Minoryx Raises $55 Million to Support MAA and Launch for X-ALD Therapy
Rare Daily Staff Spanish rare CNS disease focused biotech Minoryx Therapeutics raised $55 million (€51 million) to […]
Read moreDutch Biotech Xinvento Raises Seed Round to Develop Treatment for Congenital Hyperinsulinism
Rare Daily Staff Dutch biotech Xinvento said it completed a seed funding round, which it will use […]
Read moreHeidelberg Pharma Secures Financing Commitment from its Main Shareholder, Doses First Patient in Study
Rare Daily Staff Heidelberg Pharma said it has secured a commitment of $40.9 million (€36 million) in […]
Read moreStar Emerges from Stealth with Plans to Be “Innovation Engine” for Rare Disease Therapies, Spins out Electra
Rare Daily Staff Star Therapeutics has emerged from stealth mode with $100 million in funding today to […]
Read moreSpliceBio Raises $57 Million to Advance Protein Splicing Platform and Expand Gene Therapy Pipeline
Rare Daily Staff Spanish biotech SpliceBio completed an oversubscribed $57 million (€50 million) series A financing to […]
Read moreNMD Pharma Raises $39.7 Million to Advance Pipeline Targeting Rare Neuromuscular Disorders
Rare Daily Staff Danish biotech NMD Pharma, a clinical stage company developing first in class, small molecule […]
Read moreAlbireo Study Highlighting the Caregiver Impact of Rare Children’s Liver Disease
Rare Daily Staff Albireo Pharma said its PICTURE study evaluating and quantifying the impact that progressive familial […]
Read moreMore Stories
Magenta Therapeutics to Explore Strategic Alternatives
Rare Daily Staff Magenta Therapeutics said it has halted further development of its programs and will conduct […]
Read moreFDA Tells Taysha It Should Increase the Number of Patients in GAN Gene Therapy Study
Rare Daily Staff Taysha Gene Therapies, in providing an update on its program for giant axonal neuropathy, […]
Read moreQuince Therapeutics Prioritizes Pipeline, Slashes Workforce
Rare Daily Staff Less than a year after ditching a failed Alzheimer’s program and focusing on a […]
Read moreEMA Amends Policy of Orphan Designations of Therapies for Inherited Retinal Dystrophies
Rare Daily Staff The European Medicines Agency’s Committee on Orphan Medicinal Products said it has amended its […]
Read moreNovo Nordisk to Acquire Forma, Expanding Presence in Sickle Cell Disease and Rare Blood Disorders
Rare Daily Staff Novo Nordisk and Forma Therapeutics said that they have entered into a definitive agreement […]
Read moreFDA Approves Imbruvica for Treatment of Children with Chronic Graft Versus Host Disease
Rare Daily Staff The U.S. Food and Drug Administration approved the use of AbbVie’s Imbruvica for the […]
Read moreWhat’s Happening
What I Learned About Intrahepatic Cholestasis With Both of My Pregnancies
I frequently wake up thinking of how lucky we were. Before my first pregnancy, I have never […]
Read moreA New, More Inclusive Membership Model for Global Advocacy Alliance Members
Eligibility for Global Genes grants Access to dedicated GAA member meetings at the Annual Summit and other […]
Read moreRARE Global Advocacy Alliance Member Spotlight: Sonny Mullen
Read moreRare Diseases: Reconciling Uncertainty and Family; Leah Sarah Peer, Medical Student and third place winner of 2022 Cox Scholarship
Read moreTalking Can Be Excellent Medicine; Jenna Kwiecinski, Medical Student and second place winner of 2022 Cox Scholarship
Read moreMedical Dignity at Every Turn: Insights Learned from Rare Disease; Raymond So, Medical Student and first place winner of 2022 Cox Scholarship
Read moreRARE Global Advocacy Alliance Member Spotlight: Giovi Moschoudis
Read moreRARE Global Advocacy Alliance Member Spotlight: David Simon Ross
Read moreWhat We Learned at RARE Health Equity Summit
Read moreMore Resources: Dictionary of Rare Diseases
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