Latest Rare Diseases News

Neuren Pharmaceuticals has secured a pivotal FDA Type C Meeting to discuss primary efficacy endpoints for its Phase 3 trial of NNZ-2591 targeting Phelan-McDermid syndrome, advancing its rare disease drug development.

PYC Therapeutics has secured Safety Review Committee approval to escalate dosing in its clinical trial for Autosomal Dominant Optic Atrophy, marking a pivotal step in developing a treatment for this currently untreatable blinding disease.

Percheron Therapeutics has terminated its phase IIb avicursen trial for Duchenne muscular dystrophy after failing to meet efficacy endpoints, while securing $14.85 million in fresh capital to explore new therapeutic opportunities.