The science behind screening for Tay-Sachs disease

Shari Ungerleider lost her son to Tay-Sachs disease and spent the last 27 years working...

Canadian family raised $3.5M to develop individualized gene therapy for son’s rare condition

The family is cautiously optimistic about the improvements they are seeing in Michael, 4, since...

Lysosomes and their role in Alzheimer’s disease, Duchenne Muscular Dystrophy, Tay-Sachs Disease

U–M researchers reveal how a mutated protein acts as risk factor in Parkinson’s disease Reviewed...

Parents spark breakthrough gene therapy for devastating child disease ignored by big pharma

Exclusive: For more than a century there has been no cure, no treatment and no...

First gene therapy for Tay-Sachs disease successfully given to two children

Two babies have received the first-ever gene therapy for Tay-Sachs disease after over 14 years...

Medical Journal Publication – AAV gene therapy for Tay-Sachs Disease.

Tay-Sachs disease (TSD) is an inherited neurological disorder caused by deficiency of hexosaminidase A (HexA)....

UMass Chan launches Translational Institute for Molecular Therapeutics

Study supported by Blu Genes Published in Nature Medicine Blu Genes Foundation made a commitment...

A family from Marquette is bringing awareness to Tay-Sachs disease

MARQUETTE, Mich. (WLUC) – A rare disease for a one of a kind little boy....

Girl with genetic condition in race against time | CTV News

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An experimental gene therapy was little Alissa’s only hope. Now, instead of certain death, she faces an uncertain future.

BOSTON — Thomas Feldborg and Daria Rokina set off nearly every afternoon to explore this...