A Long-term Continuation Study of Patients with Infantile-Onset Pompe Disease who were previously enrolled in Protocol AGLU01602 A Long-term Continuation Study of Patients with Infantile-Onset Pompe Disease who were previously en ...
A Long-term Continuation Study of Patients with Infantile-Onset Pompe Disease who were previously enrolled in Protocol AGLU01602 A Long-term Continuation Study of Patients with Infantile-Onset Pompe Disease who were previously en ...
Pompe disease is a rare metabolic muscle disease inherited in an autosomal recessive fashion. Pompe disease is caused by a deficiency of GAA, which is needed for the degradation of lysosomal glycogen. Pompe disease is characterized by organelle bound (lysosomal) accumulation of glycogen in many body tissues. In general, there is an inverse correlation between the amount of residual GAA activity in patients with Pompe disease and the severity of the disease. Pompe disease is a rare metabolic muscle disease inherited in an autosomal recessive fashion. Pompe ...