Publications by authors named "Beth Leiro"

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Feasibility, Acceptability, and Reliability of Remote Motor Assessment in Children With Canavan Disease.
Michael Kiefer, Anzalee Khan, Beth Leiro, Christian Yavorsky, Genevieve Laforet

Pediatr Neurol· March 2025

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Urine -Acetylaspartate Distinguishes Phenotypes in Canavan Disease.
Amanda Nagy, Florian Eichler, Annette Bley, Janna Bredow, Alexander Fay, Beth Leiro

Hum Gene Ther· January 2024

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Challenges and strategies for clinical trials in propionic and methylmalonic acidemias.
Jerry Vockley, Barbara Burton, Agnieszka Jurecka, Jitendra Ganju, Beth Leiro

Mol Genet Metab· July 2023

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Lifelong impact of ENPP1 Deficiency and the early onset form of ABCC6 Deficiency from patient or caregiver perspective.
Christine O'Brien, Gus Khursigara, Pedro Huertas, Beth Leiro, Liz Molloy

PLoS One· July 2022

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Mucopolysaccharidosis type VI (Maroteaux-Lamy syndrome): defining and measuring functional impacts in pediatric patients.
Beth Leiro, Dawn Phillips, Melanie Duiker, Paul Harmatz, Sharon Charles

Orphanet J Rare Dis· December 2021

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Clinical Outcome Assessments: Use of Normative Data in a Pediatric Rare Disease.
Dawn Phillips, Beth Leiro

Value Health· May 2018

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Physical therapy management of infants and children with hypophosphatasia.
Dawn Phillips, Laura E Case, Donna Griffin, Kim Hamilton, Sergio Lerma Lara, Beth Leiro

Mol Genet Metab· September 2016

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