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)

Title
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)
Authors
Keywords
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Journal
MOLECULAR GENETICS AND METABOLISM
Volume 102, Issue 1, Pages 6-12
Publisher
Elsevier BV
Online
2010-09-18
DOI
10.1016/j.ymgme.2010.09.004

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