2025-11-21 • globenewswire.com
Gene Therapy for Ocular Rare Disease Market to Exhibit Phenomenal Growth During the Forecast Period (2025–2034) Due to Expanding Pipeline and Clinical Trial Activities
2025-11-21 • globenewswire.com
Gene Therapy for Ocular Rare Disease Market to Exhibit Phenomenal Growth During the Forecast Period (2025–2034) Due to Expanding Pipeline and Clinical Trial Activities2025-11-21 • PR Newswire
The Critical Need to Address Mental Health in the Rare Disease Community, Upcoming Webinar Hosted by Xtalks2025-11-20 • PR Newswire
Oxford-Harrington Rare Disease Centre Announces 2025 Scholars Advancing Promising Treatments2025-11-20 • businesswire.com
YolTech Therapeutics Receives FDA IND Clearance to Initiate Global Pivotal Trial of In Vivo Gene-Editing Therapy YOLT-203 for Primary Hyperoxaluria Type 1 (PH1)2025-11-19 • pulse2.com
Avanzanite Bioscience: €32 Million Series A Raised To Accelerate Rare Disease Expansion Across Europe2025-11-17 • quiverquant.com
Benitec Biopharma Inc. Receives FDA Fast Track Designation for BB-301 Following 100% Responder Rate in Clinical Trial2025-11-08 • musculardystrophynews.com
DMD drugs show promise despite trial’s missing main goal: Sarepta2025-11-08 • globenewswire.com
Rhythm Pharmaceuticals Announces FDA Extension of Review Period for IMCIVREE® (setmelanotide) for Patients with Acquired Hypothalamic Obesity2025-11-08 • biospace.com
Astria Therapeutics Announces Final Positive Results from All Enrolled HAE Patients in the ALPHA-STAR Phase 1b/2 Trial of Navenibart2025-11-07 • thepharmaletter.com
Ultragenyx sells portion of Crysvita royalties for $400 million2025-11-07 • globenewswire.com
Solid Biosciences Awarded Innovation Passport Designation Under the New UK Innovative Licensing and Access Pathway for SGT-003, an Investigational Gene Therapy for Duchenne Muscular Dystrophy2025-11-07 • appliedclinicaltrialsonline.com
FDA Approves UCB’s Kygevi for TK2 Deficiency Based on Multi-Study Clinical Program Demonstrating Significant Survival and Motor Gains2025-11-06 • medcitynews.com
Despite Phase 3 Failure, Sarepta Still Sees Path for Muscle Disease Drugs to Secure Full FDA Approval2025-11-06 • pharmexec.com
FDA Approves Kygevvi Becoming the First Treatment for Thymidine Kinase 2 Deficiency2025-11-06 • globenewswire.com
Palvella Therapeutics Announces New QTORIN™ Product Candidate, QTORIN™ Pitavastatin, for the Treatment of Disseminated Superficial Actinic Porokeratosis (DSAP), a Rare, Chronic, and Pre-Cancerous Genetic Skin Disease with No FDA-Approved Therapies2025-11-05 • endpoints.news
UK regulators look to speed up how rare disease therapies reach the market2025-11-04 • PR Newswire
LeMed Specialty Pharmacy and Free Market Health Partner to Accelerate Access to Rare Disease Therapies2025-11-04 • cancerhealth.com
Bizengri Shows Clinical Benefit in Rare Bile Duct Cancer2025-11-01 • medcitynews.com
Liver Complications Lead FDA to Stop Tests of Intellia Gene-Editing Therapy for a Rare Disease2025-11-01 • curetoday.com
FDA Grants Orphan Drug Designation to DPTX3186 Therapy in Gastric Cancer2025-11-01 • clinicaltrialsarena.com
Ultragenyx doses first subject in study of GTX-102 for angelman syndrome2025-10-31 • bizjournals.com
BridgeBio lands another rare disease drug trial, seeks FDA approval2025-10-31 • manilatimes.net
Ultragenyx Announces First Patient Dosed in Aurora Study Evaluating GTX-102 in Additional Angelman Syndrome Genotypes and Age Groups2025-10-31 • stocktitan.net
Ultragenyx Announces First Patient Dosed in Aurora Study Evaluating GTX-102 in Additional Angelman Syndrome Genotypes and Age Groups2025-10-29 • angioedemanews.com
HAE therapy ADX-324 hits Phase 3 milestone with first treatment2025-10-28 • biopharmadive.com
BridgeBio to seek approval of limb-girdle drug following new study data2025-10-28 • manilatimes.net
Longeveron® Co-Founder and Chief Science Officer Dr. Joshua Hare Interviewed on NPR’s BioTech Nation Discussing Potential Breakthrough Stem Cell Therapy for Hypoplastic Left Heart Syndrome (HLHS), a Rare Pediatric Congenital Heart Defect2025-10-28 • globenewswire.com
BridgeBio Reports Positive Phase 3 Results for Small Molecule BBP-418 in LGMD2I/R9 FORTIFY Study2025-10-27 • PR Newswire
Every Patient Matters: Bespoke Digital Endpoint Strategies for Rare Disease Drug Development, Upcoming Webinar Hosted by Xtalks2025-10-25 • pharmexec.com
Pharma Industry Raises Funding Aimed at Advancing the Development and Testing of Rare Disease Treatments18 hours ago • news.mongabay.com
Lesotho communities allege greenwashing by project transferring water to South Africa18 hours ago • timesofindia.indiatimes.com
SITEX 2025 to unveil cutting-edge textile tech18 hours ago • globalarbitrationreview.com
Kazakh mining investor brings ICSID claim against Guinea18 hours ago • independent.com.mt
MFSA strengthens Malta’s Islamic finance sector with new Shariah-Compliant Fund framework18 hours ago • bbc.com
Lottery funding supports new deaf charity project18 hours ago • fuelcellsworks.com
Stablegrid Group Selects ITM Power for 710 MW of Energy Infrastructure Projects in Germany19 hours ago • thestatesman.com
Ayush Ministry highlights India’s traditional healthcare legacy at Delhi Trade Fair19 hours ago • cbsnews.com
Renovations begin at Denver's Civic Center Park for the first time in 100 years19 hours ago • markets.financialcontent.com
Blue Lake Biotechnology to Resume Enrollment of RSV-negative Toddlers in Phase 1/2a Clinical Trial of BLB20119 hours ago • scanx.trade
Garg Furnace Reports Strong Q2 Performance with Focus on Value-Added Steel19 hours ago • unmannedsystemstechnology.com
Cutting-Edge Additive Manufacturing Technologies & Industrial 3D Printing Solutions for Drone & Robotics Components20 hours ago • klfy.com
Lafayette Drug Bust Finds 13k Cash Stolen Gun 170k In Drugs14 hours ago • EIN Presswire
Global Consulting Services Market Growth Outlook Strengthens Through 203313 hours ago • EIN Presswire
December Special: Free Vehicle Alignment With Any Service at 616 Auto Repair14 hours ago • PR Newswire
ModuleMD Showcases Advanced AI-Driven Allergy Solutions at ACAAI 202521 hours ago • armyrecognition.com
Russia’s Su-75 Stealth Fighter Jet Nears Bench Testing Milestone Ahead of First Flight Phase.24 hours ago • thebarentsobserver.com
New military hospital in Northern Russia opens doors for legions of wounded men2025-11-21 • defensemirror.com
UAC Delivers Improved Su-30SM2 Fighters to Russian Naval Aviation18 hours ago • timesofindia.indiatimes.com
SITEX 2025 to unveil cutting-edge textile tech18 hours ago • sentinelassam.com
Sarbananda Sonowal reviews Rs 1,000-crore water metro project across B’putra18 hours ago • fibre2fashion.com
Value shopping drives Indian festive retail, RAI reports 11% growth18 hours ago • bbc.com
Lottery funding supports new deaf charity project19 hours ago • proactiveinvestors.com
Ukog Raise More Funds As Hydrogen Project Move Forward19 hours ago • icr.ac.uk
First men invited to take part in the most ambitious prostate cancer trial in decadesStay ahead with real-time headlines from top sources, categorized by sector and country.