Canadian family raised $3.5M to develop individualized gene therapy for son’s rare condition
August 15, 2022

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
June 23, 2022

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
February 22, 2022

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
February 14, 2022

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.
February 11, 2022

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

UMass Chan launches Translational Institute for Molecular Therapeutics
February 10, 2022

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
October 8, 2021

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

Girl with genetic condition in race against time | CTV News
August 23, 2021

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An experimental gene therapy was little Alissa’s only hope. Now, instead of certain death, she faces an uncertain future.
April 30, 2021

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

‘Her body was a prison’: How a new gene therapy changed the lives of two Canadian girls
March 21, 2021

TORONTO — A new gene therapy for an extremely rare disorder has offered two Canadian...