← 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)

ICD-10 crosswalk