An open-label pilot study of losmapimod to evaluate the safety, tolerability, and changes in biomarker and clinical outcome assessments in participants with facioscapulohumeral muscular dystrophy type 1-Reference-Cited by-同舟云学术

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An open-label pilot study of losmapimod to evaluate the safety, tolerability, and changes in biomarker and clinical outcome assessments in participants with facioscapulohumeral muscular dystrophy type 1

Published:2024-07 Issue: Volume:462 Page:123096
ISSN:0022-510X
Container-title:Journal of the Neurological Sciences
language:en
Short-container-title:Journal of the Neurological Sciences

Author:

Kools Joost,Voermans Nicol,Jiang John G.,Mitelman Olga,Mellion Michelle L.,Ramana Vivekananda,van Engelen Baziel G.M.

Publisher

Elsevier BV

Reference53 articles.

1. Facioscapulohumeral muscular dystrophy;Statland;Continuum (Minneap. Minn.),2016

2. Prevalence and Incidence of Rare Diseases: Bibliographic Data;Orphanet,2022

3. Facioscapulohumeral muscular dystrophy;Statland;Neurol. Clin.,2014

4. Genetic characterization of a large, historically significant Utah kindred with facioscapulohumeral dystrophy;Flanigan;Neuromuscul. Disord.,2001

5. Population-based incidence and prevalence of facioscapulohumeral dystrophy;Deenen;Neurology,2014

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