2016
Implementing a Quality and Safety Agenda in a Children's Hospital Within a Hospital: Challenges and Successes.
Hospital pediatrics
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- Glycogen Storage Disease Type II , 6 publications
- alpha-Glucosidases , 5 publications
- Genetic Therapy , 5 publications
- Genetic Vectors , 4 publications
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16 publications
2014
Acid Alpha-Glucosidase Gene Replacement Therapy To the Diaphragm Remodels Ventilatory Function in Severe Pompe Disease
The FASEB's Journal
2014
Phase I/II trial of diaphragm delivery of recombinant adeno-associated virus acid alpha-glucosidase (rAAaV1-CMV-GAA) gene vector in patients with Pompe disease.
Human gene therapy. Clinical development
PubMed • Publisher's site2013
Acid Alpha-Glucosidase Gene Replacement Therapy To the Diaphragm in Ventilator-Dependent Pompe Disease: One-Year Respiratory Motor Outcomes
Molecular Therapy
2013
B-Cell depletion and immunomodulation before initiation of enzyme replacement therapy blocks the immune response to acid alpha-glucosidase in infantile-onset Pompe disease.
The Journal of pediatrics
PubMed • Publisher's site