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.

Authors

Rosa Puertollano¹, Nina Raben²

Affiliations

¹ Cell and Developmental Biology Center, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA.
² Cell and Developmental Biology Center, National Heart, Lung, and Blood Institute, National Institutes of Health, Bethesda, MD 20892, USA. Electronic address: rabenn@mail.nih.gov.

PMID: 34800404
DOI: 10.1016/S1474-4422(21)00358-6

No abstract available

Publication types

Research Support, Non-U.S. Gov't
Comment

MeSH terms

Enzyme Replacement Therapy
Glycogen Storage Disease Type II* / drug therapy
Humans