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FSH muscular dystrophy

Genea Biocells developed the world’s first human stem cell model of FSHD which shows cellular hallmarks of the disease. We pursue a small molecule approach to find therapeutic drug candidates that slow, stop or reverse the disease-related phenotype in skeletal muscle.  This project has been supported by the FSHD Global Research Foundation and Friends of FSH Research.

About FSHD

FSHD is a genetic disease resulting in progressive muscle weakness

Onset is typically in young adults

Affects ~1:7-8,000 people

FSHD is caused by aberrant upregulation of DUX4 – a protein toxic to muscle

For more information on FSHD visit the   → MDA website