Ontology highlight
ABSTRACT:
SUBMITTER: Cohen J
PROVIDER: S-EPMC8048701 | biostudies-literature | 2021 Feb
REPOSITORIES: biostudies-literature
Cohen Justin J DeSimone Alec A Lek Monkol M Lek Angela A
Trends in molecular medicine 20201019 2
Facioscapulohumeral muscular dystrophy (FSHD) is one of the most common types of muscular dystrophy, affecting roughly one in 8000 individuals. The complex underlying genetics and poor mechanistic understanding has caused a bottleneck in therapeutic development. Until the discovery of DUX4 and its causal role in FSHD, most trials were untargeted with limited results. Emerging approaches can learn from these early trials to increase their chance of success. Here, we explore the evolution of FSHD ...[more]