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 ...
STAT13d
Breakthrough therapies have given Duchenne muscular dystrophy families like mine hope — and new fears
Michelle C. Werner agonized over whether the gene therapy treatment Elevidys was right for her teenage son. But to him, 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 ...
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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 ...
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 ...
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 ...
Nationwide Children's names gene therapy center for retired pioneer Dr. Jerry Mendell
Nationwide Children's Hospital has renamed its gene therapy research program and clinics for Dr. Jerry Mendell, who devoted ...
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 ...
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, ...
Sarepta Therapeutics Muscles Up In DMD Race
Sarepta's Elevidys approval, revenue growth projections, and competitive position make it a strong investment in DMD ...
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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 ...
6-year-old Tennessee boy denied 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 ...