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New therapies for Pompe disease: are we closer to a cure?.

The Lancet. Neurology

Puertollano R, Raben N.
PMID: 34800404
Lancet Neurol. 2021 Dec;20(12):973-975. doi: 10.1016/S1474-4422(21)00358-6.

No abstract available.

Cite
Puertollano R, Raben N. New therapies for Pompe disease: are we closer to a cure?. Lancet Neurol. 2021;20(12):973-975doi: 10.1016/S1474-4422(21)00358-6.
Puertollano, R., & Raben, N. (2021). New therapies for Pompe disease: are we closer to a cure?. The Lancet. Neurology, 20(12), 973-975. https://doi.org/10.1016/S1474-4422(21)00358-6
Puertollano, Rosa, and Raben, Nina. "New therapies for Pompe disease: are we closer to a cure?." The Lancet. Neurology vol. 20,12 (2021): 973-975. doi: https://doi.org/10.1016/S1474-4422(21)00358-6
Puertollano R, Raben N. New therapies for Pompe disease: are we closer to a cure?. Lancet Neurol. 2021 Dec;20(12):973-975. doi: 10.1016/S1474-4422(21)00358-6. PMID: 34800404.
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Enzyme Replacement Therapy Can Reverse Pathogenic Cascade in Pompe Disease.

Molecular therapy. Methods & clinical development

Meena NK, Ralston E, Raben N, Puertollano R.
PMID: 32671132
Mol Ther Methods Clin Dev. 2020 Jun 10;18:199-214. doi: 10.1016/j.omtm.2020.05.026. eCollection 2020 Sep 11.

Pompe disease, a deficiency of glycogen-degrading lysosomal acid alpha-glucosidase (GAA), is a disabling multisystemic illness that invariably affects skeletal muscle in all patients. The patients still carry a heavy burden of the disease, despite the currently available enzyme replacement...

Cite
Meena NK, Ralston E, Raben N, et al. Enzyme Replacement Therapy Can Reverse Pathogenic Cascade in Pompe Disease. Mol Ther Methods Clin Dev. 2020;18:199-214doi: 10.1016/j.omtm.2020.05.026.
Meena, N. K., Ralston, E., Raben, N., & Puertollano, R. (2020). Enzyme Replacement Therapy Can Reverse Pathogenic Cascade in Pompe Disease. Molecular therapy. Methods & clinical development, 18199-214. https://doi.org/10.1016/j.omtm.2020.05.026
Meena, Naresh Kumar, et al. "Enzyme Replacement Therapy Can Reverse Pathogenic Cascade in Pompe Disease." Molecular therapy. Methods & clinical development vol. 18 (2020): 199-214. doi: https://doi.org/10.1016/j.omtm.2020.05.026
Meena NK, Ralston E, Raben N, Puertollano R. Enzyme Replacement Therapy Can Reverse Pathogenic Cascade in Pompe Disease. Mol Ther Methods Clin Dev. 2020 Jun 10;18:199-214. doi: 10.1016/j.omtm.2020.05.026. eCollection 2020 Sep 11. PMID: 32671132; PMCID: PMC7334420.
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Showing 13 to 14 of 14 entries