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 ...
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 ...
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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 ...
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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 Duchenne muscular dystrophy.
'The Remarkable Life of Ibelin' review: A remarkable doc about a 'World of Warcraft' player's inner life
The Remarkable Life of Ibelin blends traditional documentary techniques with World of Warcraft recreations to paint a ...
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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 ...
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12-year-old boy with muscular dystrophy banned from favourite hobby due to new rule
Brodie Pankhurst suffers from Duchenne muscular dystrophy, which progressively weakens his muscles. The cruel illness has ...
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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 ...
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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 ...
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 ...
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 ...
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Duchenne muscular dystrophy: major trials and events to watch in 2023
In 2023, two potential FDA approvals and three late-stage clinical trial readouts could shift the treatment landscape for Duchenne muscular dystrophy. The upcoming FDA approval decision for ...