7 results on '"Peña, Loren D M"'
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2. Postmortem Findings and Clinical Correlates in Individuals with Infantile-Onset Pompe Disease
3. Cardiac responses in paediatric Pompe disease in the ADVANCE patient cohort
4. Cardiac responses in paediatric Pompe disease in the ADVANCE patient cohort.
5. Contributions from medical geneticists in clinical trials of genetic therapies: A points to consider statement of the American College of Medical Genetics and Genomics (ACMG).
6. Long-term Safety and Efficacy of Avalglucosidase Alfa in Patients With Late-Onset Pompe Disease.
7. Motor Responses in Pediatric Pompe Disease in the ADVANCE Participant Cohort.
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