Duchenne muscular dystrophy

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Du·chenne muscular dystrophy

A severe form of muscular dystrophy that begins in early childhood and almost exclusively affects males, characterized by progressive weakening of the muscles and usually leading to death from respiratory or cardiac failure. It is caused by lack of the protein dystrophin in muscle cells as a result of a recessive genetic mutation on the X chromosome.

[After Guillaume B.A. Duchenne (1806-1875), French physician.]
References in periodicals archive ?
February 5, 2018 -- The FDA has granted expedited development status for an experimental cell therapy for Duchenne muscular dystrophy.
Food and Drug Administration has granted CAP-1002, its lead investigational cell therapy for the treatment of Duchenne muscular dystrophy, the Regenerative Medicine Advanced Therapy designation.
The findings are a major step towards developing a stem cell replacement therapy for muscle diseases, including Duchenne muscular dystrophy, which affects approximately one in 5,000 boys in the U.
M2 PHARMA-October 25, 2017-Capricor Presents 12-Month Results from HOPE-1 Trial in Duchenne Muscular Dystrophy
a biotechnology company developing biological therapies for Duchenne muscular dystrophy and other rare diseases, today announced that the U.
1 March 2017 - US nonprofit organisation CureDuchenne's CureDuchenne Ventures subsidiary has committed USD 5m in seed financing in Exonics Therapeutics, a new biotechnology company focused on utilising gene editing technologies like CRISPR/Cas9 to advance the development of a treatment for Duchenne muscular dystrophy, the organisation said.
Global Markets Direct's, 'Duchenne Muscular Dystrophy - Pipeline Review, H1 2016', provides an overview of the Duchenne Muscular Dystrophy pipeline landscape.
Emily Crossley, director of Duchenne Children's Trust, said: "As parents we see the benefit of steroids, but also the side-effects.
com)-- Three Duchenne muscular dystrophy nonprofits have donated $5,000 each to TREAT-NMD Advisory Committee for Therapeutics (TACT), an international expert multidisciplinary body that provides objective guidance to clinicians, researchers, patient advocacy groups and industry on advancing new therapies for neuromuscular diseases.
Bubbles' World of Play, in New Brighton, held a charity event to mark World Duchenne Day, which raises awareness and funds for charity Duchenne Now.
Simon Jameson, 27, suffers from Duchenne muscular dystrophy which causes sufferers to lose the ability to walk between the ages of 10 and 14, lose strength in their upper bodies and be left with serious heart and lung problems.
Mum-of-three Jeanette George, whose five-yearold son Alex has Duchenne muscular dystrophy, said there needed to be a more coordinated approach to services in Wales.

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