GlobalData on MSN6d
Duchenne muscular dystrophy: major trials and events to watch in 2023
A possible gene therapy approval and a series of major trials readouts set the stage for a busy year of drug development in ...
WTVC4d
6-year-old Tennessee boy denied potentially life-saving gene therapy by insurance company
Six-year-old Cooper Wood was diagnosed with Duchenne muscular dystrophy earlier this year. When his parents applied for a one ...
Doctors Said These Women’s Mutated Genes Wouldn’t Harm Them
The embryos were female, and Jenssen remembers the doctors assuring her that, because the Duchenne mutation is linked to the ...
kpax1h
Calves to Cure helps fund research for Montana boy's rare disease
The Billings Livestock Commission hosted the sixth annual Calves to Cure to help raise money and awareness for Duchenne ...
Who Is Sehba Shah? PAK Chess Player With Muscular Dystrophy Competing At Olympiad 2024
Meet Sehba Shah, Pakistan's inspiring woman chess player who lives with muscular dystrophy yet dreams high above the ...
biopharma-reporter4d
FDA designations boost AI-driven cell therapy approach for Duchenne muscular dystrophy
The FDA has granted Orphan Drug and Rare Pediatric Disease Designations to Somite’s cell replacement therapy for the ...
Business Wire2d
Neu-REFIX Beta glucan receives rare pediatric disease and orphan drug designations from the US FDA for treatment of Duchenne Muscular Dystrophy
Neu refix granted orphan disease designation (ODD) & rare pediatric disease designation (RPDD) by US FDA for treatment of ...
KCBS Radio San Francisco on MSN4d
As Prescribed: How treatment for muscular dystrophy is continuing to advance
A quarter of a million Americans are living with muscular dystrophy or a related neuromuscular disorder. It's the reason ...
cnycentral6d
6-year-old Tennessee boy denied potentially life-saving gene therapy by insurance company
ATHENS, Tenn. (WTVC) — Six-year-old Cooper Wood was diagnosed with Duchenne muscular dystrophy earlier this year. When his parents applied for a one-time gene therapy infusion that Cooper is ...
FDA backs Somite.ai's new drug for rare children's muscle disease
AI-driven biotech Somite.ai scores dual FDA designations for its novel treatment, potentially fast-tracking hope for families ...
Finger Lakes Times4d
Suneel's partners with Jett Foundation to support NY families caring for loved one with Duchenne Muscular Dystrophy
To provide hope for families grappling with Duchenne muscular dystrophy across Upstate New York, Buffalo-based Suneel’s Light Foundation has launched a new community grant program, Brighter Days Commu ...