CRISPR screen maps 250 genes essential for human muscle fiber formation

Muscles make up nearly 40% of the human body and power every move we make, from a child's first steps to recovery after ...
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 ...
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 ...
Healthline

What to Know About More Common Pediatric Musculoskeletal Disorders

Musculoskeletal disorders affect the bones, joints, muscles, and connective tissue. Many musculoskeletal disorders may occur in children, and some types are more common than others. Musculoskeletal ...
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 ...
Seeking Alpha

Atossa Therapeutics Receives FDA Rare Pediatric Disease Designation for (Z)-Endoxifen for Duchenne Muscular Dystrophy

Designation expands (Z)-Endoxifen program into rare pediatric neuromuscular disease and may qualify Atossa for a future Priority Review Voucher upon approval SEATTLE, Dec. 11, 2025 /PRNewswire/ -- ...
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 ...
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 ...