LAMA2‐related muscular dystrophy: Natural history of a large pediatric cohort

OBJECTIVE: To characterize natural history of Laminin‐α2 related muscular dystrophies (LAMA2‐RD) to help anticipating complications and identifying reliable outcome measures for clinical trial design and powering. METHODS: We conducted a retrospective, single‐center, cross‐sectional and longitudinal...

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Bibliografiske detaljer
Udgivet i:Ann Clin Transl Neurol
Main Authors: Zambon, Alberto A., Ridout, Deborah, Main, Marion, Mein, Rachael, Phadke, Rahul, Muntoni, Francesco, Sarkozy, Anna
Format: Artigo
Sprog:Inglês
Udgivet: John Wiley and Sons Inc. 2020
Fag:
Research Articles
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC7545609/
https://ncbi.nlm.nih.gov/pubmed/32910545
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/acn3.51172
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