Facioscapulohumeral Dystrophy (FSHD), also called Landouzy-Dejerine, is due to a modification of structure on a small region of chromosome 4, the D4Z4 region. It occurs with a muscular weakness affecting muscles of the face, shoulders, and arms, usually during adolescence or adulthood.
ATYR-C4 study
- Reference laboratory: Institute of Myology
This open-label, intrapatient dose escalation study aims at evaluating the safety, tolerability, immunogenicity, and biological activity of ATYR1940 in patients with limb girdle and facioscapulohumeral muscular dystrophies.
For further information: ATYR-C4 study webpage
ATYR-C3 study
- Reference laboratory: Institute of Myology
This open-label, intrapatient dose-escalation study aims at evaluating the safety, tolerability, immunogenicity, and biological activity of ATYR1940 in patients with early onset and other pediatric onset facioscapulohumeral muscular dystrophy.
For further information: ATYR-C3 study webpage
