Diego Cadavid, MD of Fulcrum Therapeutics summarizes the several abstracts that were presented at the American Academy of Neurology Annual Meeting (AAN 2020) focused on facioscapulohumeral muscular dystrophy (FSHD) and losmapimod.

FSHD is a rare progressive disease in which persons gradual lose skeletal muscle strength, initially in muscles of the face, shoulders, arms, and trunk, but later progressing to weakness throughout the lower body. FSHD is due to the mis-expression of DUX4 in skeletal muscles. Losmapimod is a selective p38α/β mitogen activated protein kinase (MAPK) inhibitor.

The six abstracts Dr. Cadavid discusses are:

  1. Phase 1 Clinical Trial of Losmapimod in Facioscapulohumeral Muscular Dystrophy (FSHD): Safety, Tolerability, and Target Engagement https://cslide-us.ctimeetingtech.com/aan2020/attendee/eposter/poster/2192
  2. Design of a Phase 2, Randomized, Double-Blind, Placebo-Controlled, 48-Week, Parallel-Group Study of the Efficacy and Safety of Losmapimod in Treating Subjects with Facioscapulohumeral Muscular Dystrophy (FSHD): ReDUX4. https://cslide-us.ctimeetingtech.com/aan2020/attendee/eposter/poster/2193
  3. Development of an Optimized Timed Up and Go (oTUG) for Measurement of Changes in Mobility Impairment in Facioscapulohumeral Muscular Dystrophy (FSHD) Clinical Trials. https://cslide-us.ctimeetingtech.com/aan2020/attendee/eposter/poster/1638
  4. A Biomarker of DUX4 Activity to Evaluate losmapimod Treatment Effect in FSHD Phase 2 Trials. https://cslide-us.ctimeetingtech.com/aan2020/attendee/eposter/poster/2196
  5. An In-Home Study of Facioscapulohumeral Muscular Dystrophy (FSHD) Patients using Contactless Wireless Sensing and Machine Learning. https://cslide-us.ctimeetingtech.com/aan2020/attendee/eposter/poster/1721
  6. Development and Evaluation of a Whole-body MRI Imaging Protocol and Analysis Algorithms to Measure Changes in Skeletal Muscle in FSHD. https://cslide-us.ctimeetingtech.com/aan2020/attendee/eposter/poster/2176

To learn more about FSHD and other rare musculoskeletal conditions, visit checkrare.com/diseases/musculoskeletal-diseases/