Business Wire

Satellos Receives Rare Pediatric Disease Designation from the U.S. FDA for SAT-3247 for the Treatment of Duchenne Muscular Dystrophy

TORONTO--(BUSINESS WIRE)--Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSX: MSCL, OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the ...
Healio

FDA grants rare pediatric disease designation for Duchenne muscular dystrophy treatment

Please provide your email address to receive an email when new articles are posted on . The FDA has granted rare pediatric disease designation to an investigational ...
Nasdaq

Edgewise Receives Orphan Drug and Rare Pediatric Disease Designations for Its Muscular Dystrophy Program from FDA

BOULDER, Colo.--(BUSINESS WIRE)-- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the U.S. Food & Drug Administration (FDA) has ...
University of Delaware

Understanding muscle development

University of Delaware’s Mona Batish is a molecular biologist. She studies circular RNAs formed as a result of so-called molecular mistakes that occur when a strand of RNA — the ribonucleic acid that ...
Tennessean

Breakthrough gene therapy offers new hope to child with muscular dystrophy

Six-year-old José Capablanca was excited to see his visitors. “I think you guys want to know how fast I can run!” he shouted, racing around his family’s home in Gallatin, Tennessee. José has Duchenne ...
Business Insider

Wave Life Sciences Receives FDA Rare Pediatric Disease Designation for WVE-N531 for the Treatment of Duchenne Muscular Dystrophy

Designation highlights significant unmet needs in DMD; dystrophin data from potentially registrational FORWARD-53 trial of WVE-N531 are on track for 3Q 2024 In previous Part A trial, WVE-N531 achieved ...
Morningstar

Precision BioSciences Receives FDA Rare Pediatric Disease Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with high unmet ...