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 ...
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 ...
PharmiWeb

Satellos Receives Clearance by U.S. FDA and Global Regulators to Initiate Pediatric Phase 2 Study of SAT-3247 for Duchenne Muscular Dystrophy

Placebo-controlled Phase 2 study will evaluate SAT-3247 treatment over three months among ambulatory children with Duchenne- Study endpoints include safety and tolerability, effect on muscle force and ...
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 ...
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 ...
Seeking Alpha

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

DURHAM, N.C.--(BUSINESS WIRE)-- Precision BioSciences, Inc. (DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for ...
News Medical

IU study sheds new light on the development and treatment of rare form of muscular dystrophy

A study by Indiana University School of Medicine researchers sheds new light on the development and treatment of a rare form of muscular dystrophy. The study's findings were recently published in ...
Seeking Alpha

CUMBERLAND PHARMACEUTICALS RECEIVES FDA ORPHAN DRUG AND RARE PEDIATRIC DISEASE DESIGNATIONS FOR NEW TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY

NASHVILLE, Tenn., Nov. 6, 2024 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (CPIX), a specialty pharmaceutical company, announced today that the United States (U.S.) Food and Drug Administration ...