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 ...
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 ...
Week In Review: Nippon Shinyaku Signs $735m Deal For DMD Therapy
Japan’s Nippon Shinyaku will acquire European rights for a Duchenne muscular dystrophy disease therapy developed by Capricor ...
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 ...
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 ...
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 ...
The American Journal of Managed Care4d
Atrial Arrhythmias Common in DMD, but More Data on Treatment Are Needed
Cases of atrial arrhythmia in patients with Duchenne muscular dystrophy (DMD) are common but lack a standard solution, and ...
KPAX Missoula, MT on MSN1d
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 ...
Somite Therapeutics Announces FDA Orphan Drug and Rare Pediatric Disease Designations for SMT-M01 in Duchenne Muscular Dystrophy
Somite Therapeutics, a fully integrated TechBio company leveraging big data and AI to introduce novel cell replacement therapies, today announced that the U.S. Food and Drug Administration (FDA) has ...
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 ...
12-year-old boy with muscular dystrophy banned from favourite hobby due to new rule
A 12-year-old boy from Sydney with a disability has been banned from participating in his favourite hobby due to a ...