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Journal of Clinical Neuromuscular Disease|December 17, 2008
Adult-Onset Presentation of Glutaric Acidemia Type II With MyopathyMichael C Mareska, Kristin K Adams, Joseph Muenzer, et al.
Ophthalmic Genetics|April 6, 2016
Homocysteinemia due to MTHFR deficiency in a young adult presenting with bilateral lens subluxationsNatario L Couser, Julie McClure, Michael W Evans, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|December 11, 2003
Self-complementary adeno-associated virus serotype 2 vector: global distribution and broad dispersion of AAV-mediated transgene expression in mouse brainHaiyan Fu, Joseph Muenzer, Richard J Samulski, et al.
Molecular Therapy. Methods & Clinical Development|September 8, 2018
Targeting Root Cause by Systemic scAAV9-h<i>IDS</i> Gene Delivery: Functional Correction and Reversal of Severe MPS II in MiceHaiyan Fu, Kim Zaraspe, Naoko Murakami, et al.
Molecular Genetics and Metabolism|February 10, 2006
Glutaric acidemia type 1 in patients of Lumbee heritage from North CarolinaAlice A Basinger, Jessica K Booker, Dianne M Frazier, et al.
Developmental Medicine and Child Neurology|September 12, 2017
Carpal tunnel syndrome in mucopolysaccharidosis I: a registry-based cohort studyDavid Viskochil, Joseph Muenzer, Nathalie Guffon, et al.
Genetics in Medicine : Official Journal of the American College of Medical Genetics|January 15, 2011
Idursulfase treatment of Hunter syndrome in children younger than 6 years: results from the Hunter Outcome SurveyJoseph Muenzer, Michael Beck, Roberto Giugliani, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|July 21, 2011
Safety and feasibility of high-pressure transvenous limb perfusion with 0.9% saline in human muscular dystrophyZheng Fan, Keith Kocis, Robert Valley, et al.
Medrxiv : the Preprint Server for Health Sciences|July 3, 2026
Differential COVID-19 Outcomes Across Lysosomal DisordersBlake K Byer, Zachary Butzin-Dozier, Brenda M McGrath, et al.
Orphanet Journal of Rare Diseases|January 7, 2021
A charitable access program for patients with lysosomal storage disorders in underserved communities worldwideAtul Mehta, Uma Ramaswami, Joseph Muenzer, et al.
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