Work

Long-term Follow-up of Cipaglucosidase Alfa/Miglustat in Ambulatory Patients with Pompe Disease: An Open-label Phase I/II Study (ATB200-02) (S48.007)

Year: 2023

Type: article

Study design: Clinical Trial

Source: Neurology

DOI: 10.1212/wnl.0000000000202282

Authors: Tahseen Mozaffar, Benedikt Schoser, Priya Kishnani, Drago Bratkovic, Paula R. Clemens, Ozlem Goker-Alpan, Xue Ming, Mark Roberts, Kumaraswamy Sivakumar, Ans Van Der Ploeg, Mitchell Goldman, Jacquelyn Wright, Fred Holdbrook, Vipul Jain, Sheela Sitaraman, Yasmine Wasfi, Barry Byrne

Institutions: University of California, Irvine, Ludwig-Maximilians-Universität München, Duke Medical Center, Royal Adelaide Hospital, University of Pittsburgh, VA Pittsburgh Healthcare System, Lysosomal and Rare Disorders Research and Treatment Center, Rutgers New Jersey Medical School, Salford Royal NHS Foundation Trust, Erasmus MC, Amicus Therapeutics (United States), University of Florida

Language: English

Topic: Lysosomal Storage Disorders Research

Subfield: Physiology

Field: Medicine

Domain: Health Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W4367305010

FWCI: 0.293

Cites:

Cited by: 2

Related to: 10