RAG-21 / Ractigen Therap - LARVOL DELTA

RAG-21-FUS-ALS: Study on the Safety and Efficacy of RAG-21 in the Treatment of Amyotrophic Lateral Sclerosis Patients With FUS Gene Mutations (clinicaltrials.gov) - P1 | N=6 | Not yet recruiting | Sponsor: Beijing Tiantan Hospital

New P1 trial • Amyotrophic Lateral Sclerosis • CNS Disorders

Ractigen Therapeutics Announces FDA Orphan Drug Designation for RAG-21 for the Treatment of ALS (PRNewswire) - "Ractigen Therapeutics...today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to RAG-21, its novel siRNA therapy targeting the FUS gene, for the treatment of amyotrophic lateral sclerosis (ALS)."

Orphan drug • Amyotrophic Lateral Sclerosis