kpax2h
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 ...
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 ...
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 ...
ktvq2d
Calves to Cure helps fund research for Worden boy's rare disease
The Billings Livestock Commission hosted the sixth annual Calves to Cure on Thursday, an event to help raise money and ...
precisionmedicineonline3d
House E&C Committee Passes Bill on Rare Pediatric Disease Drug Development
The bill's passage through the committee improves the rare pediatric disease priority review voucher program's chances of being reauthorized before it expires Sept. 30.
Muscular Dystrophy News3d
DMD treatment SMT-M01 gets FDA orphan drug, rare disease tags
DMD treatment SMT-01, a cell replacement therapy, received FDA orphan drug and rare pediatric disease designations.
Floppy limbs, weak coughs: Key warning signs of spinal muscular atrophy in babies
Delays in treatment for the rare genetic condition could lead to progressive muscle weakness and irreversible loss in motor ...
Sarepta Therapeutics Muscles Up In DMD Race
Sarepta's Elevidys approval, revenue growth projections, and competitive position make it a strong investment in DMD ...
KCBS Radio San Francisco on MSN3d
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 ...
The American Journal of Managed Care4d
Meta-Study Analyzes Immunosuppressive Protocols for AAV Gene Therapy for Monogenic Disorders
Immunosuppressants used in adeno-associated virus (AAV) gene therapy for monogenic disorders present an adverse effect ...
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 ...