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Rare disease clinical trials: Power in numbers

The limb-girdle muscular dystrophies (LGMDs) encompass a collection of genetic muscle diseases with proximal-predominant weakness of the limbs. Thirty-two of these disorders are named via the common nomenclature, including 8 autosomal-dominant (LGMD1A-H) and 24 autosomal-recessive (LGMD2A-X) disorde...

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Detalles Bibliográficos
Autor principal: Wicklund, Matthew P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Wolters Kluwer 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4974843/
https://www.ncbi.nlm.nih.gov/pubmed/27540592
http://dx.doi.org/10.1212/NXG.0000000000000092

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