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FDA delays decision on Stealth` rare genetic disorder drug
30 Apr 2025 //
REUTERS
Oxford-Harrington Rare Disease Centre Appoints Majid Jafar
29 Apr 2025 //
GLOBENEWSWIRE
DEBRA Research Invests in Nova Anchora for Epidermolysis Bullosa
29 Apr 2025 //
GLOBENEWSWIRE
EveryONE Medicines & Mendelian Partner to Transform UK Rare Disease
29 Apr 2025 //
BUSINESSWIRE
Barth Syndrome Foundation Urges FDA Action on Elamipretide Delay
29 Apr 2025 //
BUSINESSWIRE
CSL Vifor, Travere Therapeutics get EU approval for Filspari
29 Apr 2025 //
PR NEWSWIRE
Jaguar to Share Crofelemer Study Data in April 30 Webcast
29 Apr 2025 //
ACCESSWIRE
Exousia Pro Ends Glioblastoma Trial Early for Drug Filing
29 Apr 2025 //
ACCESSWIRE
US FDA approves first cell-based gene therapy for skin disorder
29 Apr 2025 //
PRESS RELEASE
Jaguar Health to Present Initial Results from Crofelemer Study
28 Apr 2025 //
ACCESSWIRE
Purespring Therapeutics granted EMA orphan drug for IgA Nephropathy
28 Apr 2025 //
GLOBENEWSWIRE
Purespring Therapeutics granted EMA orphan drug for IgAN
28 Apr 2025 //
GLOBENEWSWIRE
Argenx Announces Positive CHMP Opinion for Vygar Injection
28 Apr 2025 //
GLOBENEWSWIRE
Claritas Rx Launches Care Coordination Platform for Rare Diseases
28 Apr 2025 //
BUSINESSWIRE
Innorna gets FDA Rare Pediatric Disease, Orphan Drug Designations
27 Apr 2025 //
BUSINESSWIRE
SpringWorks Therapeutics Expects CHMP Opinion on Nirogacestat
27 Apr 2025 //
GLOBENEWSWIRE
Protara Therapeutics Reports Positive Interim Results in Phase 2
26 Apr 2025 //
GLOBENEWSWIRE
Jazz gets CHMP nod for Zanidatamab in HER2+ Biliary Tract Cancer
25 Apr 2025 //
PR NEWSWIRE
CHMP recommends Oczyesa® for acromegaly treatment in EU
25 Apr 2025 //
PR NEWSWIRE
PTC Therapeutics Gets Positive CHMP Opinion for Sephience for PKU
25 Apr 2025 //
PR NEWSWIRE
Govt exempts orphan drugs from port testing to speed up delivery
25 Apr 2025 //
ECONOMICTIMES
CHMP recommends EU approval for Duvyzat for Muscular Dystrophy
25 Apr 2025 //
GLOBENEWSWIRE
Dyne Therapeutics gets EMA Orphan Drug Designation for DYNE-251
24 Apr 2025 //
GLOBENEWSWIRE
Elafibranor Shows Safety, Efficacy in Rare Liver Disease (PSC) Trial
24 Apr 2025 //
GLOBENEWSWIRE
X4 Pharmaceuticals to Report Q1 2025 Results, Host Call on May 1
24 Apr 2025 //
GLOBENEWSWIRE
X4 Pharmaceuticals Announces Reverse Stock Split
24 Apr 2025 //
GLOBENEWSWIRE
Orphan Therapeutics Accelerator Announces Partnerships
23 Apr 2025 //
BUSINESSWIRE
X4 Pharma Announces Inducement Grants Under Nasdaq LR 5635(c)(4)
23 Apr 2025 //
GLOBENEWSWIRE
AB Science: EMA grants AB8939 orphan drug status for AML
23 Apr 2025 //
GLOBENEWSWIRE
Protara Therapeutics to Review Data Ph 2 from TARA-002 Trial
23 Apr 2025 //
GLOBENEWSWIRE
NEJM Publishes Positive Results from Insmed`s Ph 3 ASPEN Study
23 Apr 2025 //
PR NEWSWIRE
Jazz Pharmaceuticals Highlights ASCO 2025 Data on Cancer Advances
23 Apr 2025 //
PR NEWSWIRE
Viking Therapeutics Reports Q1 2025 Financial Results & Update
23 Apr 2025 //
PR NEWSWIRE
Mallinckrodt to Present Terlivaz Data at 2025 DDW Meeting
23 Apr 2025 //
PR NEWSWIRE
NeuroNOS Receives FDA Orphan Designation for Phelan-McDermid
21 Apr 2025 //
GLOBENEWSWIRE
Tempest`s TPST-1495 Granted Orphan Drug Status by FDA for FAP
21 Apr 2025 //
GLOBENEWSWIRE
Zevra Files Proxy Statement and Mails Letter to Stockholders
21 Apr 2025 //
GLOBENEWSWIRE
Immunovant announces growth phase with Roivant, leadership change
21 Apr 2025 //
GLOBENEWSWIRE
Aptevo Therapeutics $2.0M Direct Offering Priced Nasdaq Rules
21 Apr 2025 //
ACCESSWIRE
Jazz Pharmaceuticals Completes Acquisition of Chimerix
21 Apr 2025 //
PR NEWSWIRE
FDA Grants Orphan Drug Designation to NS-229
21 Apr 2025 //
PR NEWSWIRE
Roche discontinues low dose in Huntington’s trial
19 Apr 2025 //
ENDPTS
Saol Announces Clinical Milestones in Development Program
17 Apr 2025 //
PR NEWSWIRE
Caliway to Present CBL-514 Ph 2 Results at World Orphan Congress
17 Apr 2025 //
PR NEWSWIRE
uniQure`s AMT-130 Granted FDA Breakthrough Therapy for Huntington
17 Apr 2025 //
GLOBENEWSWIRE
Recordati: FDA Expands Isturisa Indication for Cushing`s Syndrome
16 Apr 2025 //
GLOBENEWSWIRE
Henlius Releases 2024 ESG Report on Human Health
16 Apr 2025 //
PR NEWSWIRE
Precigen & RRP Foundation to Host 2025 RRP Awareness Day on June
16 Apr 2025 //
PR NEWSWIRE
Spruce inks deal for BioMarin asset, plans 2026 approval filing
16 Apr 2025 //
FIERCE BIOTECH
Scilex Gets Orphan Drug Nod for Colchicine in Pericarditis
16 Apr 2025 //
GLOBENEWSWIRE
Processa Pharma` Dr. David to Present at World Orphan Congress
15 Apr 2025 //
GLOBENEWSWIRE
Er-Kim partners with Polaris Pharma for Adzodi in EMEA Market
15 Apr 2025 //
GLOBENEWSWIRE
IPAX-Linz Study Shows Promise for TLX101 Glioma Therapy Candidate
15 Apr 2025 //
PR NEWSWIRE
Norgine acquires Theravia to boost rare disease portfolio
15 Apr 2025 //
PR NEWSWIRE
n-Lorem Foundation Celebrates 5 Years Helping Nano-rare Patients
15 Apr 2025 //
BUSINESSWIRE
Sarepta Therapeutics Updates Limb-Girdle Muscular Dystrophy Programs
15 Apr 2025 //
BUSINESSWIRE
Oak Hill Bio Licenses Angelman Syndrome Treatment to Roche
15 Apr 2025 //
BUSINESSWIRE
Lobe Sciences Announces Private Placement for Drug Development
14 Apr 2025 //
ACCESSWIRE
Kedrion Biopharma Ends 2024 With 10% Growth, Rebrands
14 Apr 2025 //
PR NEWSWIRE
Sumitomo, NCI Partner to Advance Enzomenib Cancer Study
14 Apr 2025 //
PR NEWSWIRE
