Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)

Exclusion Criteria

• •serious medical illness, significant cardiac disease or severe debilitating pulmonary disease;
• •any hematologic abnormality, especially megaloblastic anemia, leukopenia, thrombocytopenia, pancytopenia;
• •any active uncontrolled bleeding or any bleeding diathesis (e.g., active peptic ulcer disease);
• •possible folate deficiency, and those receiving therapy (such as phenytoin) affecting folate levels;
• •any complex disease that may confound treatment assessment;
• •pregnant women or women of child-bearing potential not using reliable means of contraception;
• •lactating females;
• •fertile men unwilling to practice contraceptive methods during the study period;
• •unwilling or unable to follow protocol requirements;
• •known hypersensitivity reactions, intolerance or adverse reactions to pyrimethamine;
• •evidence of active infection, or serious infection within the past month;
• •HIV infection;
• •a history of cancer of any type;
• •receiving any other standard or investigational treatment for any indication within the past 4 weeks prior to initiation of pyrimethamine treatment;
• •receiving immunotherapy of any type within the past 4 weeks prior to initiation of pyrimethamine treatment; or any condition or abnormality, which may, in the opinion of the investigator, compromise the safety of patients.