Newborn Screening for Pompe Disease: Pennsylvania Experience

Míreanna comhchosúla

• Successful desensitization protocol to alglucosidase and avalglucosidase alfa in a patient with infantile-onset Pompe disease
  de réir: Miriam Gendive, et al.
  Foilsithe / Cruthaithe: (2025-06-01)
• Higher dose alglucosidase alfa is associated with improved overall survival in infantile-onset Pompe disease (IOPD): data from the Pompe Registry
  de réir: Priya S. Kishnani, et al.
  Foilsithe / Cruthaithe: (2023-12-01)
• Characteristics of Pompe disease in China: a report from the Pompe registry
  de réir: Yuying Zhao, et al.
  Foilsithe / Cruthaithe: (2019-04-01)
• Newborn Screening for Pompe Disease: Pennsylvania Experience
  de réir: Can Ficicioglu, et al.
  Foilsithe / Cruthaithe: (2020-11-01)
• Efficacy of avalglucosidase alfa on forced vital capacity percent predicted in treatment-naïve patients with late-onset Pompe disease: A pooled analysis of clinical trials
  de réir: Tahseen Mozaffar, et al.
  Foilsithe / Cruthaithe: (2024-09-01)