Kristen Truxal

A prospective one-year natural history study of mucopolysaccharidosis types IIIA and IIIB: Implications for clinical trial design

Molecular Genetics and Metabolism · 2016 · First of 15 authors · 41 citations

Differential Prevalence of Antibodies Against Adeno-Associated Virus in Healthy Children and Patients with Mucopolysaccharidosis III: Perspective for AAV-Mediated Gene Therapy

Human Gene Therapy Clinical Development · 2017 · 39 citations