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Ultragenyx Fill Ph3 Aspire Study for GTX-102 in Angelman Syndrome
31 Jul 2025 //
GLOBENEWSWIRE
AZ`s Alexion Highlights gMG in Upcoming Awareness Film
31 Jul 2025 //
FIERCE PHARMA
FDA Approves Alhemo for Hemophilia A/B Treatment in Children
31 Jul 2025 //
PR NEWSWIRE
Ph 2 Trial Shows SGX945`s Efficacy in Treating Behçet`s Disease
31 Jul 2025 //
PR NEWSWIRE
Sanofi’s SAR446523 Gets US Orphan Drug Tag For Multiple Myeloma
31 Jul 2025 //
PRESS RELEASE
Orsini Chosen as PTC Therapeutics` Sephience Pharmacy Partner
30 Jul 2025 //
PR NEWSWIRE
Vivace Therapeutics Gets Orphan Drug Designation for VT3989
30 Jul 2025 //
PR NEWSWIRE
EU Grant Orphan Status to Sumitomo`s Nuvisertib for Myelofibrosis
30 Jul 2025 //
PR NEWSWIRE
AscellaHealth & Abeona Launch Novel Gene Therapy for Rare Disease
29 Jul 2025 //
GLOBENEWSWIRE
Cognition Showcases Zervimesine`s Impact on Dementia & Alzheimer
29 Jul 2025 //
GLOBENEWSWIRE
AbbVie, Gilead, GSK Support Annual Hepatitis Elimination Drive
29 Jul 2025 //
FIERCE PHARMA
CHMP Recommends AQNEURSA For Niemann-Pick Approval In EU
29 Jul 2025 //
BUSINESSWIRE
US FDA approves PTC Therapeutics` Metabolic Disorder Drug
29 Jul 2025 //
REUTERS
Apellis snatches broad kidney Disease Expansion for Empaveli
29 Jul 2025 //
FIERCE PHARMA
Kedrion COAGADEX Gets Orphan Drug Status for aFXD, Begins Trial
29 Jul 2025 //
PR NEWSWIRE
FDA Approves Doptelet for Pediatric ITP: Sobi
25 Jul 2025 //
GLOBENEWSWIRE
AstraZeneca`s Rare Immune Disorder Drug Succeeds in Trial
25 Jul 2025 //
REUTERS
Prilenia, Ferrer Give EU Update on Pridopidine in Huntington
25 Jul 2025 //
BUSINESSWIRE
Primus Sounds Alarm on Chronic Venous Insufficiency Amid
24 Jul 2025 //
ACCESSWIRE
Medunik USA Expands Siklos® Medicaid Coverage to 37 States
22 Jul 2025 //
PR NEWSWIRE
Analysis Group Explores HEOR Use in Rare Disease CGTs
22 Jul 2025 //
PR NEWSWIRE
EU Grants Conditional Approval of Ezmekly for Patient with NF1-PN
18 Jul 2025 //
GLOBENEWSWIRE
Aardvark Therapeutics Grants Inducements Under Nasdaq Listing
18 Jul 2025 //
GLOBENEWSWIRE
Zevra Therapeutics Presents Miplyffa® and Olpruva® at SERGG
18 Jul 2025 //
GLOBENEWSWIRE
SAPU Bio`s TGFB2 Tied to Survival in Young Pancreatic Cancer
17 Jul 2025 //
GLOBENEWSWIRE
Oncotelic Therapeutic TGFB2 Linked to Pancreatic Cancer Survival
17 Jul 2025 //
GLOBENEWSWIRE
Cycle Launches Harliku Tablets for Alkaptonuria (AKU)
17 Jul 2025 //
BUSINESSWIRE
Rocket Gets FDA RMAT for RP-A601 Gene Therapy
17 Jul 2025 //
BUSINESSWIRE
Leo Ponies up €90 Million for Boehringer Ingelheim`s Spevigo
15 Jul 2025 //
BUSINESSWIRE
KalVista’s Ekterly Gets UK Nod for Hereditary Angioedema
15 Jul 2025 //
BUSINESSWIRE
AlzeCure`s ACD440 Granted Orphan Drug Status by FDA
15 Jul 2025 //
ACCESSWIRE
How to Get Pharmaceutical Treatments Where They’re Needed Most
14 Jul 2025 //
FIERCE BIOTECH
NanoViricides Nears Completion of MPox Clade I Trial Design
14 Jul 2025 //
ACCESSWIRE
FDA grants Klotho Neurosciences orphan drug status for ALS
11 Jul 2025 //
PR NEWSWIRE
Ultragenyx, Mereo Plummet After Rare Diseases Trial Result
11 Jul 2025 //
GLOBENEWSWIRE
European Commission Approve Tevimbra for Nasopharyngeal Carcinoma
10 Jul 2025 //
BUSINESSWIRE
Biogen, Stoke Reveal Zorevunersen Data for Dravet Syndrome
10 Jul 2025 //
GLOBENEWSWIRE
Arcus Biosciences’ Quemliclustat Receives Orphan Drug Designation
10 Jul 2025 //
BUSINESSWIRE
EU Establishes Expert Group for Pediatric and Rare Disease
09 Jul 2025 //
RAPS
Zhimeng Biopharma`s KCNQ2/3 Opener Approved for ALS Phase 2/3
09 Jul 2025 //
PR NEWSWIRE
IASO Bio Gets Orphan Tag In South Korea For Equecabtagene
09 Jul 2025 //
PR NEWSWIRE
Orsini to Partner as Specialty Pharmacy for KalVista`s Ekterly
07 Jul 2025 //
PR NEWSWIRE
Spinogenix`s Spg601 Granted Orphan Drug Status for Fragile X
07 Jul 2025 //
PR NEWSWIRE
Ascendis to Share Endocrinology Rare Disease Data at ENDO 2025
07 Jul 2025 //
GLOBENEWSWIRE
Immix Speeds Up NEXICART-2 Trial in AL Amyloidosis
07 Jul 2025 //
GLOBENEWSWIRE
U.S. FDA Grants Orphan Drug Designation to Mustang Bio`s MB-101
07 Jul 2025 //
GLOBENEWSWIRE
FDA accepts Denali`s BLA for Tividenofusp Alfa in Hunter Syndrome
07 Jul 2025 //
GLOBENEWSWIRE
BioCryst names Babar Ghias CFO, Head of Corporate Development
07 Jul 2025 //
GLOBENEWSWIRE
Ema Designates Bexobrutideg for Waldenström Macroglobulinemia
07 Jul 2025 //
GLOBENEWSWIRE
FDA grants Breakthrough Therapy Designation to Lexeo`s LX2006
07 Jul 2025 //
GLOBENEWSWIRE
ALS Community Urges FDA Approval of NurOwn Stem Cell Therapy
07 Jul 2025 //
ACCESSWIRE
Abeona Sells Rare Pediatric Disease Voucher for $155 Million
02 Jul 2025 //
GLOBENEWSWIRE
Belite Bio Completes Enrollment in Global Phase 3 PHOENIX Trial
02 Jul 2025 //
GLOBENEWSWIRE
Bio-Thera`s BAT4406F Gets IDMC Backing in NMOSD Trial
02 Jul 2025 //
PR NEWSWIRE
Apellis to Get $300M from Royalty Deal with Sobi for Aspaveli
01 Jul 2025 //
GLOBENEWSWIRE
Syntis Bio secures $38M to speed up oral therapies for obesity
01 Jul 2025 //
BUSINESSWIRE
Sarcoidosis Research Foundation Launches Enhanced Data Registry
01 Jul 2025 //
GLOBENEWSWIRE
Tilde Sciences Launches VECAMYL™ Through Optime Care
30 Jun 2025 //
GLOBENEWSWIRE
argenx progresses ARGX-119 for Congenital Myasthenic Syndromes
30 Jun 2025 //
GLOBENEWSWIRE
Riliprubart Wins Japan Orphan Drug Tag for CIDP Therapy
30 Jun 2025 //
PRESS RELEASE
