Article

Landscape and Heterogeneity of Treatment Outcome Measures in Facioscapulohumeral Muscular Dystrophy: A Systematic Literature Review

2025-09-02

Abstract excerpt

Facioscapulohumeral muscular dystrophy is a rare genetic disorder that manifests as progressive weakening and loss of skeletal muscles, with the face, shoulder girdle, and upper arms typically affected in early stages. Currently, there are no approved therapies, and no consensus on the outcome measures used to assess disease progression or effects of new interventions. This systematic literature review identified...

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Literature Corpus work: dcbe1599-05c6-57d7-8cc9-079fc4c1f647
DOI: 10.1101/2025.08.29.25334711

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