Pompe disease: Unmet needs and emerging therapies

Pompe disease is a debilitating and life-threatening disease caused by aberrant accumulation of glycogen resulting from reduced acid alpha-glucosidase activity. The first treatment for Pompe disease, the enzyme replacement therapy, Myozyme® (recombinant human acid alpha-glucosidase, alglucosidase al...

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Veröffentlicht in:	Molecular genetics and metabolism 2024-11, Vol.143 (3), p.108590, Article 108590
Hauptverfasser:	George, Kelly A., Anding, Allyson L., van der Flier, Arjan, Tomassy, Giulio S., Berger, Kenneth I., Zhang, Tracy Y., Sardi, S. Pablo
Format:	Artikel
Sprache:	eng
Schlagworte:	1-Deoxynojirimycin - analogs & derivatives 1-Deoxynojirimycin - therapeutic use alpha-Glucosidases - genetics alpha-Glucosidases - therapeutic use Drug development Emerging therapy Enzyme Replacement Therapy Gene therapy Genetic Therapy Glycogen - metabolism Glycogen Storage Disease Type II - drug therapy Glycogen Storage Disease Type II - therapy Humans Lysosomal storage disorders Pompe disease Substrate reduction therapy
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