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Sarcoglycanopathies

Deficiencies or mutations in the genes for the SARCOGLYCAN COMPLEX subunits. A variety of phenotypes are associated with these mutations including a subgroup of autosomal recessive limb girdle muscular dystrophies, cardiomyopathies, and respiratory deficiency.

Drugs by status

Phase 2 trials (1)

Organizations

Organization Involved with Phase 2 Indications (2)