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).
Clarke
Joe T R
JT
Research Institute, Hospital for Sick Children, Toronto, Ontario, Canada. jtrc@sickkids.ca
Mahuran
Don J
DJ
Sathe
Swati
S
Kolodny
Edwin H
EH
Rigat
Brigitte A
BA
Raiman
Julian A
JA
Tropak
Michael B
MB