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(Reuters) -A young patient died due to cardiac arrest after receiving Pfizer's experimental gene therapy being tested in a mid-stage trial for a muscle-wasting disorder called Duchenne muscular ...
The therapy, Elevidys, was granted accelerated approval in June last year to treat Duchenne muscular dystrophy, an inherited progressive muscle-wasting disorder that almost always affects young boys.
Duchenne muscular dystrophy is a rare progressive disease that eventually affects all voluntary muscles and involves the heart and breathing muscles in later stages. Life expectancy is estimated to be around 25–26, [18] [59] but this varies. People born with Duchenne muscular dystrophy after 1990 have a median life expectancy of approximately ...
In May 2014, ataluren received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) [18] and received market authorization from the European Commission to treat people with nonsense mutation Duchenne muscular dystrophy in August 2014; [2] a confirmatory phase III clinical trial ...
Primary mitochondrial myopathies are inherited, while secondary mitochondrial myopathies may be inherited (e.g. Duchenne's muscular dystrophy) [3] or environmental (e.g. alcoholic myopathy [4] [5]). When it is an inherited primary disease, it is one of the metabolic myopathies. [6] [4]
My 11-year-old son has Duchenne muscular dystrophy, a rare disease with no cure. But finding the words to tell him the truth wasn't easy.
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