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Implementation of a neuromuscular clinical trial network: a rare disease model for enhancing clinical trial readiness, capacity, and access in Canada

Abstract Background Rare diseases affect over 300 million people globally, yet clinical trial conduct in rare disease populations remains complex due to small patient numbers, geographic dispersion, heterogeneous phenotypes, and limited trial infrastructure. Neuromuscular diseases (NMDs) exemplify t...

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Autors principals: Kerri Lynn Schellenberg, Homira Osman, Maria Masnata, Rhiannon Hicks, Corinne Kagan, Ana Stosic, Stacey Lintern, Erin Beattie, Hanns Lochmuller, Craig Campbell, Jean K. Mah
Format: Artigo
Idioma:Inglês
Publicat: BMC 2026-05-01
Col·lecció:Orphanet Journal of Rare Diseases
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Accés en línia:https://doi.org/10.1186/s13023-026-04393-4
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