← All indications
Glycogen Storage Disease Type VII
An autosomal recessive glycogen storage disease in which there is deficient expression of 6-phosphofructose 1-kinase in muscle (PHOSPHOFRUCTOKINASE-1, MUSCLE TYPE) resulting in abnormal deposition of glycogen in muscle tissue. These patients have severe congenital muscular dystrophy and are exercise intolerant.
Drugs by status
Phase 2 trials (1)
Other trials (1)
Organizations
Organization Involved with Phase 2 Indications (2)