The first child-specific body scan charts could enable physicians to assess obesity risk

A Concordia-led research team has created one of the first reference charts showing how children's adipose (fat) and muscle ...
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 ...
Everyday Health

Does My Child Have Duchenne Muscular Dystrophy?

Find out if it’s time to ask about your child’s symptoms — and if they could be a sign of Duchenne. Duchenne muscular dystrophy — sometimes referred to simply as Duchenne — is a genetic disorder that ...
Business Wire

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 ...
MedPage Today

Kids with Spinal Muscular Atrophy Rate Their Own Health

"Our preliminary findings show that German children with SMA, despite significant physical disability, have surprisingly good subjective HRQoL." – Erik Landfeldt, MSc, PhD Previous studies have found ...
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 ...