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
FDA Approves Gamifant for MAS in Still’s Disease Patients
27 Jun 2025 //
PR NEWSWIRE
Schrödinger Gets Fast Track for SGR-1505 in Macroglobulinemia
27 Jun 2025 //
BUSINESSWIRE
Ultragenyx Earns Breakthrough Status for GTX-102 in Angelman
27 Jun 2025 //
GLOBENEWSWIRE
Orphalan partners with MAP International and Wilson Disease Assoc
27 Jun 2025 //
GLOBENEWSWIRE
Scinai Immuno’s PC111 Wins Top Italian Dermatology Award
26 Jun 2025 //
PR NEWSWIRE
Grace Therapeutics Submits New Drug Application for GTx-104
25 Jun 2025 //
GLOBENEWSWIRE
Ovid Therapeutics Enters Agreement with Immedica Pharma
25 Jun 2025 //
GLOBENEWSWIRE
FDA Grants RPD Tag to QRX003 for Netherton Syndrome
24 Jun 2025 //
GLOBENEWSWIRE
PharmaTher Secures Key US Patent for Ketamine in ALS
24 Jun 2025 //
GLOBENEWSWIRE
Abeona Ph 3 RDEB Data Published in The Lancet Journal
24 Jun 2025 //
GLOBENEWSWIRE
Jaguar Health Extends Maturity Date of Convertible Notes to Jan
24 Jun 2025 //
ACCESSWIRE
Jaguar Health Updates Orphan Disease Intestinal Failure Program
23 Jun 2025 //
ACCESSWIRE
Cumberland Pharmaceuticals Shares Phase 2 FIGHT DMD Trial Results
23 Jun 2025 //
PR NEWSWIRE
Sephience (Sepiapterin) Approved by European Commission for PKU
23 Jun 2025 //
PR NEWSWIRE
Cure Rare Disease Gets FDA Feedback on LGMD2I Gene Therapy
23 Jun 2025 //
BUSINESSWIRE
Arcturus Therapeutic to Host KOL Presentation on ARCT-810 Phase 2
23 Jun 2025 //
BUSINESSWIRE
Dupixent US Approval For Bullous Pemphigoid Treatment
21 Jun 2025 //
PRESS RELEASE
SpringWorks Therapeutics gets CHMP nod for Nirogacestat
20 Jun 2025 //
GLOBENEWSWIRE
Cycle Pharma Harliku Tablets FDA-Approved for Alkaptonuria
20 Jun 2025 //
BUSINESSWIRE
UCB presents TK2d research at UMDF 2025 Conference
20 Jun 2025 //
PR NEWSWIRE
Larimar Sets Call on Nomlabofusp for Friedreich’s Ataxia
20 Jun 2025 //
GLOBENEWSWIRE
Argenx gets EU approval for Vyvgart injection for CIDP
20 Jun 2025 //
GLOBENEWSWIRE
Capricor Therapeutics: Positive 4-Year Data from HOPE-2 Study
20 Jun 2025 //
GLOBENEWSWIRE
Saol Therapeutics Poster at UMDF Mitochondrial Med. Conf. 2025
19 Jun 2025 //
PR NEWSWIRE
DEA Judge Exposes Bias in Cannabis Grower Licensing Case
19 Jun 2025 //
ACCESSWIRE
Chiesi Global Rare Diseases Awards Research Grants for Lysosomal
19 Jun 2025 //
BUSINESSWIRE
Paradigm Therapeutics Receives Investment to Develop SD-101
18 Jun 2025 //
PR NEWSWIRE
Camurus` POSITANO study: CAM2029 effects in liver disease
18 Jun 2025 //
PR NEWSWIRE
BPGbio Presents Preliminary Clinical Data for BPM31510
18 Jun 2025 //
GLOBENEWSWIRE
Senti Bio`s SENTI-202 Granted FDA Orphan Drug Designation for AML
18 Jun 2025 //
GLOBENEWSWIRE
Diagonal’s DIAG723 Antibody Gets ODD from U.S. FDA and EMA
18 Jun 2025 //
GLOBENEWSWIRE
Telomir Pharmaceuticals Prevents Cellular Aging in Progeria
18 Jun 2025 //
ACCESSWIRE
JCR Pharmaceuticals Launches MPS Type II Disease Awareness Film
18 Jun 2025 //
BUSINESSWIRE
Actio Biosciences Raises $66M for Genetics-Driven Therapeutics
18 Jun 2025 //
BUSINESSWIRE
FDA Grants Orphan Designation To Medicovestor ADC For Cancer
17 Jun 2025 //
GLOBENEWSWIRE
Capricor Gets Orphan Drug Designation For BMD, Advances DMD
17 Jun 2025 //
GLOBENEWSWIRE
Lobe Advances Psilocin After Spinout Deal With Cynaptec
17 Jun 2025 //
ACCESSWIRE
RemeGen`s Telitacicept (RC18) Received Orphan Drug Designation
17 Jun 2025 //
PR NEWSWIRE
Azitra Reports Positive Ph1b Data In Netherton Syndrome
17 Jun 2025 //
PR NEWSWIRE
Sarepta Updates ELEVIDYS Safety for Non-Ambulatory DMD
16 Jun 2025 //
BUSINESSWIRE
US FDA approves Nuvation Bio`s rare lung cancer drug
12 Jun 2025 //
REUTERS
Angitia Biopharmaceuticals Announces Topline Results of AGA2115
12 Jun 2025 //
GLOBENEWSWIRE
Novartis Fabhalta® improves hemoglobin in new PNH patients
12 Jun 2025 //
GLOBENEWSWIRE
Blueprint Medicines: Data on AYVAKIT® Treatment at 2025 Congresse
12 Jun 2025 //
PR NEWSWIRE
EU Commission Grants Orphan Drug Status to Florbetaben (18F)
12 Jun 2025 //
PR NEWSWIRE
Sumitomo Pharma America: Nuvisertib (TP-3654) Gets FDA Fast Track
12 Jun 2025 //
PR NEWSWIRE
Vascarta gets FDA Orphan Drug Designation for Vasceptor
12 Jun 2025 //
PR NEWSWIRE
Mezzion Expands Udenafil Pipeline to Target ADPKD Kidney Market
12 Jun 2025 //
PR NEWSWIRE
Ouro Medicines to Trial OM336 for Autoimmune Cytopenias
12 Jun 2025 //
PR NEWSWIRE
Sobi shares new clinical data on hematologic diseases at EHA 2025
12 Jun 2025 //
PR NEWSWIRE
Incyte’s Retifanlimab Phase 3 Data in SCAC Published in Lancet
12 Jun 2025 //
BUSINESSWIRE
Ionis CDO Dr Geary to Retire Dr Kordasiewicz Named Successor
12 Jun 2025 //
BUSINESSWIRE
ENYO Pharma Completes Series C Financing with Vesalius Biocapital
12 Jun 2025 //
BUSINESSWIRE
Travere to Present Filspari Data at Podocyte Conference
11 Jun 2025 //
BUSINESSWIRE
First Patient Dosed in Ph 3 Study of ION582 in Angelman syndrome
11 Jun 2025 //
BUSINESSWIRE
Avidity Biosciences to Present Phase 1/2 Fortitude Trial Data
11 Jun 2025 //
PR NEWSWIRE
FDA Grants Orphan Drug Tag to Veverimer for Anti-GBM
10 Jun 2025 //
PR NEWSWIRE
Vanda Pharmaceuticals: First Patient Dosed in VCA-894A Trial
10 Jun 2025 //
PR NEWSWIRE