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FDA Approves Arrowhead`s Genetic Disorder Drug
19 Nov 2025 //
FDA
Stoke, Biogen Publish 2-Year Dravet Data
18 Nov 2025 //
BUSINESSWIRE
Transcon CNP Trial In Children With Achondroplasia Published
17 Nov 2025 //
GLOBENEWSWIRE
Nusinersen High Dose Regimen Gets Positive CHMP Opinion
17 Nov 2025 //
GLOBENEWSWIRE
Bioinvent`s BI?1808 Receives EMA Orphan Drug Designation
17 Nov 2025 //
PHARMIWEB
Thermo Fisher Unveils Corevitas Registry For Myasthenia Gravis
17 Nov 2025 //
BUSINESSWIRE
FDA Fast-Tracks Dewpoint`s DPTX3186 to Treat Gastric Cancer
17 Nov 2025 //
GLOBENEWSWIRE
US FDA Grants Orphan Drug Designation To OBI-902
17 Nov 2025 //
PHARMIWEB
Dawnzera Receives Positive Opinion from CHMP
15 Nov 2025 //
BUSINESSWIRE
FDA Limits Elevidys Use Due To Fatal Liver Injury Reports
15 Nov 2025 //
GLOBENEWSWIRE
Ascelia Pharma Submits Orviglance NDA To FDA For Review
15 Nov 2025 //
ACCESSWIRE
Telethon`s Waskyra Gene Therapy Gets Positive CHMP Opinion
14 Nov 2025 //
PR NEWSWIRE
Analysts Skeptical Applied Can Bring Rare Disease Drug to Market
14 Nov 2025 //
FIERCE BIOTECH
FDA Narrows Elevidys Duchenne Gene Therapy Patient Pool
14 Nov 2025 //
FDA
NHS Scotland Recommends Procysbi For Nephropathic Cystinosis
13 Nov 2025 //
PHARMIWEB
Chiesi Enters Into License Agreement with Aliada Therapeutics
13 Nov 2025 //
GLOBENEWSWIRE
FDA Unveils New Pathway To Speed Custom Gene Editing Therapies
13 Nov 2025 //
FIERCE BIOTECH
Eton Pharma To Attend 16th Craig-Hallum Alpha Select Conference
12 Nov 2025 //
GLOBENEWSWIRE
Neurogene Reports Positive Data from NGN-401 Gene Therapy Trial
12 Nov 2025 //
BUSINESSWIRE
Servier India Introduces Affordable Biomarker Tests For Rare
12 Nov 2025 //
BUSINESS STD
Calliditas Reports Data On Setanaxib In Alport Syndrome at ASN
11 Nov 2025 //
PR NEWSWIRE
Innate Pharma Announces FDA Clearance to Proceed With TELLOMAK 3
10 Nov 2025 //
BUSINESS WIRE
FDA Grants Orphan Status To Tinostamustine For Malignant Glioma
10 Nov 2025 //
BUSINESS WIRE
Quince Therapeutics Announces Positive iDSMB Review for eDSP
10 Nov 2025 //
BUSINESS WIRE
Rhythm Pharma Reveals Four Datasets At Obesityweek Conference
10 Nov 2025 //
GLOBENEWSWIRE
Regeneron, AZ Regulatory Plans Bolstered By Ph. 3 Wins
07 Nov 2025 //
BUSINESSWIRE
FDA Approves Kygevvi for TK2d in Adults & Children
07 Nov 2025 //
PR NEWSWIRE
Mirum Highlights LIVMARLI And Volixibat Data At Liver Meeting
07 Nov 2025 //
BUSINESSWIRE
FDA Extends Review For IMCIVREE In Acquired Hypothalamic Obesity
07 Nov 2025 //
GLOBENEWSWIRE
Biocryst Reveals Orladeyo`s Sustained Efficacy In Pediatric HAE
06 Nov 2025 //
GLOBENEWSWIRE
Ionis Reveals Dawnzera`s Long-Term Disease Control At ACAAI
06 Nov 2025 //
BUSINESSWIRE
Zydus Gets FDA Orphan Drug Designation For Desidustat
06 Nov 2025 //
INDPHARMAPOST
Neurelis to Present Study of Nrl-1049 at Annual CCM Meeting
06 Nov 2025 //
PR NEWSWIRE
Neurogene Initiates NGN-401 Gene Therapy Trial For Rett Syndrome
06 Nov 2025 //
BUSINESSWIRE
Solid Biosciences Receives UK Innovation Designation For SGT-003
06 Nov 2025 //
GLOBENEWSWIRE
MHRA Commits To Major Reform Of Rare Disease Therapy Regulation
06 Nov 2025 //
RAPS
FDA Rejects Biohaven Approval Request, Triggering Cost Cutting
06 Nov 2025 //
BIOSPACE
Calico Gets FDA Orphan Tag For ADPKD Investigational Drug
05 Nov 2025 //
PR NEWSWIRE
First-Of-Its-Kind Cream For Chronic Hand Eczema Patients On NHS
05 Nov 2025 //
PHARMIWEB
Palvella Unveils Qtorin Pitavastatin For DSAP Treatment
05 Nov 2025 //
GLOBENEWSWIRE
Polyactiva, Raresight Partner On Rare Pediatric Retinal Disease
05 Nov 2025 //
BUSINESSWIRE
Rezolute Plans Virtual Investor Event For Ersodetug Development
05 Nov 2025 //
GLOBENEWSWIRE
Rhythm Pharma Launches IMCIVREE Reimbursement In Canada
05 Nov 2025 //
GLOBENEWSWIRE
FDA Designates Leukogene`s M2T-CD33 For Acute Myeloid Leukemia
05 Nov 2025 //
BUSINESSWIRE
Sarepta, Despite Key Trial Flop, Will Seek Full Nod For DMD Meds
04 Nov 2025 //
FIERCE PHARMA
Medunik USA Enhances Pheburane Medicaid Coverage
04 Nov 2025 //
PR NEWSWIRE
AGAMREE (Vamorolone) Shows Safer Profile In DMD Patients
04 Nov 2025 //
PHARMIWEB
Recordati Rare Diseases To Present New Data at ASH Meeting
04 Nov 2025 //
PR NEWSWIRE
Ultragenyx Sells Future Crysvita Royalties For $400 Million
04 Nov 2025 //
GLOBENEWSWIRE
Baylor Genetics Highlights Rare Disease Testing At NSGC 2025
04 Nov 2025 //
GLOBENEWSWIRE
Agios To Unveil Mitapivat Data In Rare Blood Disorders
03 Nov 2025 //
GLOBENEWSWIRE
Capricor Publishes Study on Deramiocel MOA and Potency Assay
03 Nov 2025 //
GLOBENEWSWIRE
Chiesi, Protalix Seek EMA Re-Exam for Elfabrio 4-Week Dosing
03 Nov 2025 //
GLOBENEWSWIRE
Fulcrum Therapeutics To Unveil Pociredir Data In Sickle Cell
03 Nov 2025 //
GLOBENEWSWIRE
Pierre Fabre Pharma Acquires Tabelecleucel BLA For EBV+ PTLD
03 Nov 2025 //
PR NEWSWIRE
Viridian Therapeutics Submits Veligrotug (BLA) For Thyroid Eye
03 Nov 2025 //
BUSINESSWIRE
Belite Bio Gets Conditional UK Approval For Tinlarebant
02 Nov 2025 //
GLOBENEWSWIRE
BOOSTB4 Trial Data Revealed At Osteogenesis Imperfecta Conference
31 Oct 2025 //
GLOBENEWSWIRE
Servier`s Voranigo Wins 2025 Prix Galien USA Award
31 Oct 2025 //
PR NEWSWIRE
Abeona Pharma Secures Permanent J-Code for Zevaskyn
30 Oct 2025 //
GLOBENEWSWIRE
