Establishing how much improvement in lung function and distance walked is clinically important for adult patients with Pompe disease

Documenti analoghi

• Start, switch and stop (triple‐S) criteria for enzyme replacement therapy of late‐onset Pompe disease: European Pompe Consortium recommendation update 2024
  di: Benedikt Schoser, et al.
  Pubblicazione: (2024)
• Improving outcome measures in late onset Pompe disease: Modified Rasch‐Built Pompe‐Specific Activity scale
  di: Harmke A. van Kooten, et al.
  Pubblicazione: (2024)
• Bulbar muscle impairment in patients with late onset Pompe disease: Insight from the French Pompe registry
  di: Emilie Retailleau, et al.
  Pubblicazione: (2024)
• Reply to “Reconsidering Central and Peripheral Contributions to Propranolol's Antitremor Effects in Parkinson's Disease”
  di: Anouk van der Heide, et al.
  Pubblicazione: (2025)
• Causes of Death and Comorbidities in Adult Patients With Late‐Onset Pompe Disease: A French Pompe Registry Retrospective Study
  di: Diana Maria Chitimus, et al.
  Pubblicazione: (2025)