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Study Objectives: Assessment of the safety of diaphragm administration of a recombinant adeno-associated virus gene vector, rAAV1-CMV-GAA (labeled as rAAV1-p43.2-hGAA), in children with Pompe disease(More)
Pompe disease is an inherited neuromuscular disease caused by deficiency of lysosomal acid alpha-glucosidase (GAA) leading to glycogen accumulation in muscle and motoneurons. Cardiopulmonary failure(More)