International Pompe Association

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2013

Impact of enzyme replacement therapy on survival in adults with Pompe disease: results from a prospective international observational study
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 (ABSTRACT ONLY)
 
 
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