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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) Download

Canavan Gene Therapy - Gao Final Report Download

Canavan gene therapy - Gao Progress report Download

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

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

Effective gene therapy in an authentic model of Tay-Sachs related diseases Download

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

Funded Research Projects Download

Gene Therapy Nature Biotech Letter to the Editor Download

Gene Transfers Corrects Acute GM2 Gangliosidosis-Potential Therapeutic Contribution of Perivascular Enzyme Flow Download

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