Treatment and Specialist Treatment Centres in the UK for Pompe disease
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Pompe disease (glycogen storage type II disease) is caused by a deficiency of acid alphaglucosidase (GAA) enzyme activity, resulting in lysosomal glycogen accumulation in muscles and irreversible muscle damage.1-3
Website with information for patients prescribed Nexviadyme
MAT-XU-2305069 (v1.0) Date of preparation: November 2023