Harmke A. van Kooten

Safety and efficacy of avalglucosidase alfa versus alglucosidase alfa in patients with late-onset Pompe disease (COMET): a phase 3, randomised, multicentre trial

Jordi Díaz‐Manera, Priya S. Kishnani, Hani Kushlaf et al. · The Lancet Neurology · 2021 · 184 citations · Full text

Veterinary Medicine, Internal Medicine, Pharmacotherapy +3

Efficacy and Safety of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease After 97 Weeks

Priya S. Kishnani, Jordi Díaz‐Manera, António Toscano et al. · JAMA Neurology · 2023 · 55 citations · Full text

Life Sciences, Internal Medicine, Pharmacotherapy +8

Discontinuation of enzyme replacement therapy in adults with Pompe disease: Evaluating the European POmpe Consortium stop criteria

Harmke A. van Kooten, Laurike Harlaar, Nadine A. M. E. van der Beek et al. · Neuromuscular Disorders · 2019 · 18 citations · Full text

Pharmacy, Pharmacology, Pharmacotherapy +17

Distal muscle weakness is a common and early feature in long-term enzyme-treated classic infantile Pompe patients

Jan J. A. van den Dorpel, Esther Poelman, Laurike Harlaar et al. · Orphanet Journal of Rare Diseases · 2020 · 15 citations · Full text

Medicine, Pediatrics, Neuromuscular Disorders +12

Chest MRI to diagnose early diaphragmatic weakness in Pompe disease

Laurike Harlaar, Pierluigi Ciet, Gijs van Tulder et al. · Orphanet Journal of Rare Diseases · 2021 · 14 citations · Full text

Medicine, Neurology, Radiology +16