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LGMDD1 natural history and phenotypic spectrum: Implications for clinical trials

OBJECTIVE: To delineate the full phenotypic spectrum and characterize the natural history of limb girdle muscular dystrophy type D1 (LGMDD1). METHODS: We extracted age at clinical events of interest contributing to LGMDD1 disease burden via a systematic literature and chart review. Manual muscle tes...

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Detalles Bibliográficos
Autores principales:	Findlay, Andrew R., Robinson, Sarah E., Poelker, Stephanie, Seiffert, Michelle, Bengoechea, Rocio, Weihl, Conrad C.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2022
Materias:	Research Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9930420/ https://www.ncbi.nlm.nih.gov/pubmed/36427278 http://dx.doi.org/10.1002/acn3.51709

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