Enzyme replacement therapy with alglucosidase alfa in Pompe disease: Clinical experience with rate escalation.
- Ankit K Desai,
- Crista K Walters,
- Heidi L Cope,
- Zoheb B Kazi,
- Stephanie M DeArmey,
- Priya S Kishnani
Mol Genet Metab 2018 Feb 23;123(2):92-96. Epub 2017 Dec 23.
Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USA. Electronic address: