The clinical relevance of outcomes used in late-onset Pompe disease: can we do better?
Author:
Publisher
Springer Science and Business Media LLC
Subject
Pharmacology (medical),Genetics(clinical),General Medicine
Reference69 articles.
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1. Improving outcome measures in late onset Pompe disease: Modified Rasch‐Built Pompe‐Specific Activity scale;European Journal of Neurology;2024-08-28
2. Extensive mobile health technology assessment detects subtle motor impairment in mild and asymptomatic Pompe disease;2024-07-03
3. Minimal clinically important differences in six-minute walking distance in late-onset Pompe disease;Orphanet Journal of Rare Diseases;2024-04-11
4. Walking test outcomes in adults with genetic neuromuscular diseases: a systematic literature review of their measurement properties;European Journal of Physical and Rehabilitation Medicine;2024-04
5. Real-world outcomes from a series of patients with late onset Pompe disease who switched from alglucosidase alfa to avalglucosidase alfa;Frontiers in Genetics;2024-01-19
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