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Documents, publications, and other information relating to research of allied diseases.

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An open-label Phase I/II clinical trial of pyrimethamine for the treatment of patients affected with chronic GM2 gangliosidosis (Tay–Sachs or Sandhoff variants)

Canavan gene therapy - Gao Progress report

Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?

Dramatic Phenotypic Improvement after Adeno-Associated Virus Gene Therapy in a Feline Model of Sandhoff Disease

Eichler 12 month progress report Feb 2012

Eichler 12 month Progress Report “Clinical Outcome Measures for Gene Therapy Trial in Infantile and Juvenile GM2”

h-hexosaminidase lentiviral vectors: transfer into the CNS via systemic administration

Short Description:: Brain inflammation in GM2 gangliosidosis has been recently realized as a key factor in disease development. The aim of this study was toinvestigate...

Late-onset Tay–Sachs disease: Adverse effects of medications and implications for treatment - Shapiro et al.

Long-Term Phenotypic Correction of Feline Lysosomal Storage Disease by Intracranial AAV Gene Therapy

Lysosomal Storage Disease Research Consortium Grants

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