Association of Miglustat With Swallowing Outcomes in Niemann-Pick Disease, Type C1

IMPORTANCE: Niemann-Pick disease, type C1 (NPC1) is a progressive neurovisceral disease with no US Food and Drug Administration–approved therapy. Miglustat, a drug used off-label in the United States for the treatment of NPC1, appears to stabilize neurologic disease progression. Several prospective...

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Dades bibliogràfiques
Publicat a:JAMA Neurol
Autors principals: Solomon, Beth I., Smith, Andrew C., Sinaii, Ninet, Farhat, Nicole, King, Monique C., Machielse, Leonza, Porter, Forbes D.
Format: Artigo
Idioma:Inglês
Publicat: American Medical Association 2020
Matèries:
Brief Report
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC7489403/
https://ncbi.nlm.nih.gov/pubmed/32897301
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1001/jamaneurol.2020.3241
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