The Pompe Registry: tracking Pompe disease symptoms in a broad patient population

Eitemau Tebyg

• Development of a disease severity scoring system for patients with Pompe disease
  gan: Giannini, E, et al.
  Cyhoeddwyd: (2008)
• GAA variants and phenotypes among 1,079 patients with Pompe disease: Data from the Pompe Registry
  gan: Reuser, Arnold J. J., et al.
  Cyhoeddwyd: (2019)
• Antibody formation to enzyme therapy in classic infantile Pompe disease: implications of patient age
  gan: van Gelder, C, et al.
  Cyhoeddwyd: (2013)
• Pompe disease- experience from South India
  gan: Lingappa, Lokesh, et al.
  Cyhoeddwyd: (2013)
• Impact of enzyme replacement therapy on survival in adults with Pompe disease
  gan: Güngör, Deniz, et al.
  Cyhoeddwyd: (2013)