Rare Diseases

BioNJ BioPartnering Conference: Biotech Innovation on Display

biotech · 8 min

Palvella Therapeutics Begins Phase 2 Trial of QTORIN Rapamycin for Rare Angiokeratomas

2 min

Opus Genetics' OPGx-LCA5 Gene Therapy Accepted Into FDA's Rare Disease Evidence Program

FDA · 2 min

Clene Receives FDA Green Light for CNM-Au8 Accelerated Approval Pathway in ALS Treatment

FDA · 2 min

Takeda's TAK-881 Shows Positive Phase 2/3 Results for Primary Immunodeficiency Disease Treatment

2 min

Clene's CNM-Au8 Gets FDA Green Light for Accelerated Approval Pathway in ALS Treatment

FDA · 2 min

Atossa Therapeutics Receives FDA Rare Pediatric Disease Designation for (Z)-Endoxifen in McCune-Albright Syndrome

FDA · 2 min

Novartis Receives Health Canada Approval for Fabhalta Oral Treatment for C3G Patients

2 min

Mirum Pharmaceuticals VISTAS Study Results for Volixibat in Primary Sclerosing Cholangitis Due May 4, 2026

2 min

NIKTIMVO (Axatilimab) Approved in Australia as First-in-Class Chronic Graft-Versus-Host Disease Treatment

2 min

Neurocrine Biosciences CRENESSITY Shows Sustained Benefits in Two-Year Pediatric CAH Study

2 min

ARCHIMED Acquires Esperion Therapeutics in Strategic Cardiometabolic Deal

cardiometabolic and rare/orphan diseases · 3 min