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PathGennie Speeds Up Rare Molecular Event Simulations
31 Dec 2025 //
EXPRESSPHARMA
NEJM Publishes Denali`s DNL310 Phase 1/2 Hunter Study
30 Dec 2025 //
GLOBENEWSWIRE
Medcare UAE Treat 1st International SMA Patient With Gene Therapy
30 Dec 2025 //
BUSINESSWIRE
Ultragenyx Submits BLA to FDA for DTX401 Therapy in GSDIa
30 Dec 2025 //
GLOBENEWSWIRE
Zevra Therapeutics Expands MIPLYFFA Access For Niemann-Pick
29 Dec 2025 //
GLOBENEWSWIRE
Ultragenyx Reports Setrusumab (UX143) Phase 3 Results
29 Dec 2025 //
GLOBENEWSWIRE
Mereo Biopharma Reveals Setrusumab (UX143) Phase 3 Results
29 Dec 2025 //
GLOBENEWSWIRE
Chugai`s Tecentriq Approved In Japan For Thymic Carcinoma
27 Dec 2025 //
INDPHARMAPOST
Italfarmaco, JCR Pharma Partner On Givinostat Commercialization
24 Dec 2025 //
BUSINESSWIRE
SN BioScience Gains FDA Orphan Drug Status for SNB-101
24 Dec 2025 //
PR NEWSWIRE BIO
Leading Biotech: Incentives Vital for Rare Disease Drugs
23 Dec 2025 //
PHARMIWEB
FDA Approves Agios` AQVESME For Anemia In Adult Thalassemia
23 Dec 2025 //
GLOBENEWSWIRE
EU Approves Sanofi`s Wayrli as First BTK Inhibitor for ITP
23 Dec 2025 //
PRESS RELEASE
FDA Approves JASCAYD Tablets For Progressive Pulmonary Fibrosis
22 Dec 2025 //
PR NEWSWIRE
PTC Therapeutics Gets Japan Approval For Sephience In (PKU)
22 Dec 2025 //
PR NEWSWIRE BIO
Savara Resubmits BLA for Molbreevi to Treat Autoimmune PAP
22 Dec 2025 //
BUSINESSWIRE
Ipsen Fails to Land Pivotal Rare Disease Trial
20 Dec 2025 //
FIERCE BIOTECH
Biomarin to Buy Amicus for $4.8 B in Rare Disease Bet
19 Dec 2025 //
PRESS RELEASE
US FDA Approves Cytokinetics` Heart Disease Drug
19 Dec 2025 //
PRESS RELEASE
Mandos, Subsidiary Of Beren, Submits Adrabetadex NDA
19 Dec 2025 //
PHARMIWEB
FDA Approves JASCAYD Tablets For Progressive Pulmonary Fibrosis
19 Dec 2025 //
GLOBENEWSWIRE
FDA Approves Cytokinetics` MYQORZO for Symptomatic Obstructive
19 Dec 2025 //
GLOBENEWSWIRE
Kyowa Kirin Poised For Windfall With Newborn Screening Update
19 Dec 2025 //
BIOSPACE
Sanofi`s Efdoralprin Alfa Granted EU Orphan Designation
18 Dec 2025 //
PRESS RELEASE
Memo Therapeutics AG`s Potravitug Receives Orphan Designation
18 Dec 2025 //
GLOBENEWSWIRE
Otsuka Chooses Pantherx Rare For VOYXACT Distribution
18 Dec 2025 //
PR NEWSWIRE
SGX945 Phase 2 Trial Shows Promising Results In Behçet`s Disease
18 Dec 2025 //
PR NEWSWIRE
FSR Releases Patient Voice Report Post-FDA PFDD Meeting
18 Dec 2025 //
GLOBENEWSWIRE
Addition Debuts $100M Fund for Rare Disease One-Time Therapies
17 Dec 2025 //
BIOSPACE
Hansa Updates Pivotal Phase 3 Anti-GBM Disease Trial
17 Dec 2025 //
PHARMIWEB
Ionis Ramps Up Angelman Syndrome Awareness With Lifetime Segment
17 Dec 2025 //
FIERCE PHARMA
Sol-Gel Updates On SGT-210 Phase 1B Study In Darier Disease
17 Dec 2025 //
GLOBENEWSWIRE
Soligenix Announces Top-line Results of the Ph 2a Study of SGX302
17 Dec 2025 //
PR NEWSWIRE
Orchard Celebrates Addition of Metachromatic Leukodystrophy
16 Dec 2025 //
GLOBENEWSWIRE
FDA Fast-Tracks Palvella`s QTORIN for Angiokeratomas
16 Dec 2025 //
GLOBENEWSWIRE
U.S. HHS Adds DMD to Recommended Newborn Screening Panel
16 Dec 2025 //
GLOBENEWSWIRE
Ambros Launches With $125M To Bring Italian Non-Opioid Pain Drug
16 Dec 2025 //
PR NEWSWIRE
Kennedy Approves Adding Two Rare Disorders to Newborn Screenings
16 Dec 2025 //
REUTERS
Capricor & Parent Project Muscular Dystrophy Host Webinar
16 Dec 2025 //
GLOBENEWSWIRE
Chiesi Global Rare Diseases Receives Canada Approval of Elfabrio
16 Dec 2025 //
GLOBENEWSWIRE
Immusoft Secures FDA Orphan Status for ISP-002 in MPS II
15 Dec 2025 //
PR NEWSWIRE
Jaguar Health Announces Abstract Submission for Preliminary Data
15 Dec 2025 //
PHARMIWEB
Telethon Pharma, Orphan Therapeutics Accelerator Ink Pact
15 Dec 2025 //
PR NEWSWIRE
Quince Therapeutics Completes Phase 3 NEAT Trial
15 Dec 2025 //
BUSINESSWIRE
Sentynl Therapeutics Announces FDA Acceptance Of CUTX-101 NDA
15 Dec 2025 //
PR NEWSWIRE
JAMA Acknowledges GUARDIAN Newborn Genomic Screening Study
15 Dec 2025 //
BUSINESSWIRE
Fortress Biotech, Cyprium Therapeutics Announce FDA Acceptance
15 Dec 2025 //
GLOBENEWSWIRE
CIRM Funds $7.4M for LGMD2I/R9 Therapy Development
15 Dec 2025 //
BUSINESSWIRE
Roxadustat Gains FDA Orphan Status for Myelodysplastic Syndromes
15 Dec 2025 //
GLOBENEWSWIRE
Acadia Pharma Gets FDA Nod For DAYBUE STIX In Rett Syndrome
12 Dec 2025 //
BUSINESSWIRE
Biocryst Launches ORLADEYO Oral Pellets For Pediatric HAE
12 Dec 2025 //
GLOBENEWSWIRE
Sanofi’s Qfitlia and Cablivi approved in China
12 Dec 2025 //
PRESS RELEASE
Rezolute Stock Sinks 87% As Hypoglycemia Drug Fails Ph. 3 Test
12 Dec 2025 //
FIERCE BIOTECH
Orphalan Announces Acquisition Of Orphelia Pharma
12 Dec 2025 //
PR NEWSWIRE
Sobi Gets CHMP Nod For Aspaveli In C3G And Primary IC-MPGN
12 Dec 2025 //
PR NEWSWIRE
Xinnate Gets FDA Nod For TCP-25, Advancing Epidermolysis Bullosa
11 Dec 2025 //
PHARMIWEB
Abeona Therapeutics Sets up Zevaskyn Treatment Center in Texas
11 Dec 2025 //
GLOBENEWSWIRE
FDA Approves Uplizna for Generalized Myasthenia Gravis in Adults
11 Dec 2025 //
PR NEWSWIRE
Atossa Pharma Gets FDA Rare Pediatric Disease Status For DMD
11 Dec 2025 //
PR NEWSWIRE
Ophirex Nears FDA Approval for Varespladib Snakebite Drug
11 Dec 2025 //
GLOBENEWSWIRE
