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.
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 ...
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 ...
FierceBiotech4d
Capricor sells Europe rights to DMD therapy for $35M as FDA application approaches
Having already scooped up the U.S. rights to Capricor Therapeutics’ late-stage Duchenne muscular dystrophy (DMD) therapy, ...
Benitec Biopharma CEO to Participate in OPMD Awareness Day Webinar
I am honored to participate in the inaugural OPMD Awareness Day Webinar along with patients, patient advocates, and a clinician expert, to provide an update on the BB-301 Phase 1b/2a clinical ...