In a groundbreaking study, researchers at McMaster University have identified a potential treatment for Sandhoff and Tay-Sachs diseases—two rare, often fatal lysosomal storage disorders that cause ...

Watching someone go through this is heartbreaking.” Tay-Sachs disease, the more common of the two disorders, typically manifests within the first year of life, progressing quickly and often ...

Watching someone go through this is heartbreaking." Tay-Sachs disease, the more common of the two disorders, typically manifests within the first year of life, progressing quickly and often ...

Watching someone go through this is heartbreaking." Tay-Sachs disease, the more common of the two disorders, typically manifests within the first year of life, progressing quickly and often ...

In the study, published in the journal Human Molecular Genetics, the team tested 4-PBA in a mouse model of the disease. The results showed that 4-PBA significantly improved motor function, extended ...

Genetic counselors interpret family and medical histories to assess the chance of disease occurrence or recurrence, educate families about inheritance, management, prevention, resources and research, ...

A team of Canadian researchers has found an existing US Food and Drug Administration-approved drug that could significantly ...

Common Heart Drug May Slow Progression of Huntington's Disease Dec. 2 ... have identified a potential treatment for Sandhoff and Tay-Sachs diseases -- two rare, often fatal lysosomal storage ...

Most diagnoses occur when newborns are tested, but if you have a family history, you should seek genetic counseling and prenatal genetic testing. Tay-Sachs disease Tay-Sachs is a rare inherited brain ...

sickle cell disease, Tay-Sachs disease, and phenylketonuria (PKU). X-linked dominant disorders are caused by mutations in genes on the X (female) chromosome. Females have two X chromosomes and will ...

“Fact Checking” with the Patient Community: An Industry-Advocacy Collaboration for Optimizing Trial Design: This poster is ...