Lentiviral vectors (LV) are widely used vehicles for gene transfer and therapy in pre-clinical
animal models and clinical trials with promising safety and efficacy results. However, host …

Pompe disease is a rare inherited metabolic disorder of defective lysosomal glycogen
catabolism due to a deficiency in acid alpha-glucosidase (GAA). Alglucosidase alfa enzyme …

Glycogen storage disease type II or Pompe disease is a severe neuromuscular disorder
caused by mutations in the lysosomal enzyme, acid α-glucosidase (GAA), which result in …

Objective To determine the effects of 10 years of enzyme replacement therapy (ERT) in adult
patients with Pompe disease, focusing on individual variability in treatment response …

Pompe disease is an autosomal recessive disorder caused by a deficiency of acid alpha-
glucosidase resulting in intralysosomal glycogen accumulation in multiple tissue types …

Pompe disease (PD) is a neuromuscular disorder caused by acid α‐glucosidase (GAA)
deficiency. Reduced GAA activity leads to pathological glycogen accumulation in cardiac …

Pompe disease (PD) is a monogenic disorder caused by mutations in the acid alpha-
glucosidase gene (Gaa). GAA is a lysosomal enzyme essential for the degradation of …

Enzyme replacement therapy (ERT) is the current standard treatment for Pompe disease, a
lysosomal storage disorder caused by deficiency of the lysosomal enzyme acid alpha …

Adeno-associated virus (AAV) vectors are promising candidates for gene therapy and have
been explored as gene delivery vehicles in the treatment of Duchenne Muscular Dystrophy …

Background: Pompe disease is a lysosomal storage disease characterised by skeletal and
respiratory muscle weakness. Since 2006, enzyme replacement therapy (ERT) with …