Kennedy Looks to Fast-Tracking Approvals for Rare Disease Drugs
06 Jun 2025 //
REUTERS
Dyne Therapeutics to Present FSHD Data at FSHD Society Congress
05 Jun 2025 //
GLOBENEWSWIRE
Avadel Gets Orphan Drug Nod for Lumryz in Hypersomnia
05 Jun 2025 //
GLOBENEWSWIRE
Acadia Pharmaceuticals to Host R&D Day in NYC on June 25, 2025
05 Jun 2025 //
BUSINESSWIRE
FDA’s Prasad Vows to Make Rare Disease Drugs Available
05 Jun 2025 //
ENDPTS
ViGeneron Rebrands, Gains FDA RPDD for VG801 in Stargardt
05 Jun 2025 //
GLOBENEWSWIRE
Tempest`s Amezalpat gets Orphan Drug Designation in EU for HCC
05 Jun 2025 //
GLOBENEWSWIRE
Oncovita`s MVdeltaC gets FDA Orphan Drug Designation
04 Jun 2025 //
GLOBENEWSWIRE
Quince Therapeutics Exceeds 75% Enrollment in Phase 3 NEAT Trial
03 Jun 2025 //
BUSINESSWIRE
Affinia Therapeutics and DCM Foundation Partner to Raise Awarenes
03 Jun 2025 //
BUSINESSWIRE
Rilzabrutinib Grants Orphan Designation for Sickle Cell Disease
03 Jun 2025 //
PRESS RELEASE
Sanofi to buy US biopharma group Blueprint for over $9 billion
03 Jun 2025 //
PRESS RELEASE
Ainos` AI Nose Accuracy Breakthrough in Elderly Care Monitoring
03 Jun 2025 //
ACCESSWIRE
Lobe Sciences Names Yong Yao Chief Financial Officer
03 Jun 2025 //
ACCESSWIRE
Spinogenix`s SPG302 for ALS Gets EMA Orphan Drug Designation
03 Jun 2025 //
PR NEWSWIRE
uniQure Updates on AMT-130 for Huntington`s Disease
02 Jun 2025 //
GLOBENEWSWIRE
Protagonist & Takeda`s ASCO Presentation on Ph 3 VERIFY Study
01 Jun 2025 //
BUSINESSWIRE
J Ints Bio to Present Global Clinical Results at ASCO 2025
01 Jun 2025 //
PR NEWSWIRE
LBL-024 Clinical Data in Advanced EP-NEC Revealed at 2025 ASCO
31 May 2025 //
PR NEWSWIRE
Entrada Therapeutics Authorized for ELEVATE-45-201 Study in EU
29 May 2025 //
GLOBENEWSWIRE
Ainos AI Nose Boosts Hygiene Monitoring Accuracy to 85%
29 May 2025 //
ACCESSWIRE
Zydus gets USFDA Fast Track for oral inhibitor Usnoflast
29 May 2025 //
INDPHARMAPOST
Stealth to Cut Jobs After FDA Rejects Therapy for Rare Disorder
29 May 2025 //
REUTERS
Barth Syndrome Foundation Responds to FDA`s Elamipretide Plan
29 May 2025 //
BUSINESSWIRE
Twymeeg Data Presented at 68th Japan Diabetes Society Meeting
28 May 2025 //
BUSINESSWIRE
CARsgen`s Satri-cel Granted Priority Review by the NMPA
28 May 2025 //
PR NEWSWIRE
New Therapy for Children with Growth Hormone Deficiency Approved
28 May 2025 //
PR NEWSWIRE
Taysha Gene Reveals TSHA-102 Trial Design for Rett Syndrome
28 May 2025 //
GLOBENEWSWIRE
Rezolute Completes Enrollment in Phase 3 Study of Ersodetug
28 May 2025 //
GLOBENEWSWIRE
DEBRA, Dermaliq Partner On Epidermolysis Bullosa Drug Delivery
28 May 2025 //
GLOBENEWSWIRE
Biohaven Announces Positive Data for BHV-1400 in IgA Nephropathy
28 May 2025 //
PR NEWSWIRE
Sobi to share new clinical data and research at EULAR 2025
28 May 2025 //
PR NEWSWIRE
Acadia Pharmaceuticals Appoints Allyson as Senior Vice President
27 May 2025 //
BUSINESSWIRE
Alligator Bioscience gets EU Orphan Drug Designation for HLX22
27 May 2025 //
ACCESSWIRE
Ambry Genetics Program Impact on Rare Disease Diagnosis
27 May 2025 //
BUSINESSWIRE
Moderna to Present at Upcoming Conferences in June 2025
27 May 2025 //
ACCESSWIRE
Grin Therapeutics, Angelini Pharma collaborate on Radiprodil
27 May 2025 //
PR NEWSWIRE
Rein Therapeutics Begins RENEW Phase 2 Trial of LTI-03 in IPF
27 May 2025 //
PR NEWSWIRE
Spur Shows Gene Therapy Proof for FLT201 in Gaucher Diseases
27 May 2025 //
GLOBENEWSWIRE
Ocugen Gets Rare Disease Designation For OCU410ST Gene Therapy
27 May 2025 //
GLOBENEWSWIRE
Three Factors to Make a Next-Gen eCOA Platform Work
23 May 2025 //
EXPRESSPHARMA
SpringWorks Therapeutics gets CHMP approval for Mirdametinib
23 May 2025 //
GLOBENEWSWIRE
England`s NICE Recommends Filspari for IgA Nephropathy
23 May 2025 //
PR NEWSWIRE
Innate Pharma Reports Durable Lacutamab Results
23 May 2025 //
BUSINESSWIRE
Relief Gets FDA Rare Pediatric Tag For RLF-TD011
22 May 2025 //
ACCESSWIRE
Soleno Therapeutics Submits EMA Application for Diazoxide Choline
22 May 2025 //
GLOBENEWSWIRE
Quoin Pharmaceuticals gets FDA clearance for QRX003 study
22 May 2025 //
GLOBENEWSWIRE
Merck Showcases Innovation in Advanced Cancers and Rare Tumors
22 May 2025 //
BUSINESSWIRE
PacBio Expands Distribution in China, Accesses New Lab Networks
22 May 2025 //
GLOBENEWSWIRE
Telomir Pharmaceutical Raises $3M in Equity Sale for Rare Disease
21 May 2025 //
ACCESSWIRE
Dr. Benjamin Greenberg Joins Trethera Scientific Advisory Board
21 May 2025 //
GLOBENEWSWIRE
AnnJi Pharma Reports Positive Phase 1/2a results for AJ20
21 May 2025 //
PR NEWSWIRE
Sarepta Updates UK Dosing in Elevidys Study for DMD
21 May 2025 //
BUSINESSWIRE
Tempus, Verastem Team on CDx for KRAS-Mutant Ovarian Cancer
20 May 2025 //
BUSINESSWIRE
Jaguar Starts Phase 2 Trial of Crofelemer for Pediatric MVID
20 May 2025 //
ACCESSWIRE
Soleno: VYKAT XR Re-Treatment Eases Prader-Willi Symptoms
20 May 2025 //
GLOBENEWSWIRE
Quoin Pharma EMA Grants Orphan Drug Designation for QRX003
20 May 2025 //
GLOBENEWSWIRE
SIFI gets Marketing Authorisation for Akantior® in UK
19 May 2025 //
PR NEWSWIRE
Applied`s rare disease drug suffers another phase 3 failure
19 May 2025 //
FIERCE BIOTECH
Ionis chalks up phase 3 trial win as it works to grow Tryngolza
19 May 2025 //
FIERCE PHARMA