Company to begin enrollment of patients aged 1-3 years Expects data from younger cohort to be part of pivotal plans and BLA filing for broad label End-of-Phase II meeting with FDA scheduled for late July to finalize pivotal program design Based on recent commercial...
Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), applauds the U.S. Food and Drug Administration (FDA) for its decision to expand the labeled indication for ELEVIDYS, a micro-dystrophin...
On June 6th, Moderna announced that its investigational therapy for methylmalonic acidemia (MMA), mRNA-3705, has been chosen for the FDA’s Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program. First launched in September 2023, the...
Tiziana Life Sciences Ltd. (Nasdaq: TLSA) (“Tiziana” or the “Company”), a biotechnology company developing breakthrough immunomodulation therapies via novel routes of drug delivery, today announced it has received acceptance of its submission for intranasal foralumab...
The START pilot program aims to accelerate the development of novel drug and biological products for rare diseases rAAV-Olig001-ASPA was selected as one of a few CBER-regulated products based on eligibility criteria, including clinical benefit for rare diseases with...
