Phase I/II Trial of Adeno-Associated Virus–Mediated Alpha-Glucosidase Gene Therapy to the Diaphragm for Chronic Respiratory Failure in Pompe Disease: Initial Safety and Ventilatory Outcomes

Lignende værker

• Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in Children Affected by Pompe Disease
  af: Corti, Manuela, et al.
  Udgivet: (2017)
• Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning
  af: Corti, Manuela, et al.
  Udgivet: (2015)
• Pompe disease gene therapy
  af: Byrne, Barry J., et al.
  Udgivet: (2011)
• Diaphragm Pacing as a Rehabilitative Tool for Patients With Pompe Disease Who Are Ventilator-Dependent: Case Series
  af: Smith, Barbara K., et al.
  Udgivet: (2016)
• Altered Activation of the Diaphragm in Late-Onset Pompe Disease
  af: Smith, Barbara K., et al.
  Udgivet: (2015)