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Article

The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials.

Journal of neurology, neurosurgery, and psychiatry - 1 Feb 2016

Ricotti Valeria, Ridout Deborah A, Pane Marika, Main Marion, Mayhew Anna, Mercuri Eugenio, Manzur Adnan Y, Muntoni Francesco

Abstract excerpt

OBJECTIVE: With the emergence of experimental therapies for Duchenne muscular dystrophy (DMD), it is fundamental to understand the natural history of this disorder to properly design clinical trials. The aims of this study were to assess the effects produced on motor function by different DMD genotypes and early initiation of glucocorticoids. METHODS: Through the NorthStar Network, standardised clinical data...

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Identifiers

PMID: 25733532
DOI: 10.1136/jnnp-2014-309405
PMCID: PMC4752678

Genes

DMD

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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