Symptoms and impacts of ambulatory nonsense mutation Duchenne muscular dystrophy: a qualitative stud...
Symptoms and impacts of ambulatory nonsense mutation Duchenne muscular dystrophy: a qualitative study and the development of a patient-centred conceptual model
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Cham: Springer International Publishing
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English
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Cham: Springer International Publishing
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Background
Duchenne muscular dystrophy is a rare genetic neuromuscular disorder, which can result in early death due to disease progression. Ataluren is indicated for the treatment of nonsense mutation Duchenne muscular dystrophy, in ambulatory individuals aged two years and older. This study explored the symptoms and impacts of nonsense mutatio...
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Symptoms and impacts of ambulatory nonsense mutation Duchenne muscular dystrophy: a qualitative study and the development of a patient-centred conceptual model
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TN_cdi_doaj_primary_oai_doaj_org_article_7f1f9d55effd4cc5815d2a4924c107ec
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_7f1f9d55effd4cc5815d2a4924c107ec
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ISSN
2509-8020
E-ISSN
2509-8020
DOI
10.1186/s41687-021-00341-x
