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Article

Oligonucleotide Therapies for Facioscapulohumeral Muscular Dystrophy: Current Preclinical Landscape

2024-07-22

Abstract excerpt

Facioscapulohumeral muscular dystrophy (FSHD) is an inherited myopathy, characterized by progressive and asymmetric muscle atrophy, primarily affecting muscles of the face, shoulder girdle, and upper arms before affecting muscles of the lower extremities with age and greater disease severity. FSHD is a disabling condition, and patients may also present with various extramuscular symptoms. FSHD is caused by the abe...

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Literature Corpus work
d3bb8554-f0b2-586e-bfdc-b1fb79ec8b2d
DOI
10.20944/preprints202407.1712.v1
Open publication

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