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...

SickKids researchers ‘model’ future of precision medicine for rare inherited disorders
March 19, 2021

A team of researchers at The Hospital for Sick Children (SickKids) has successfully implemented a...

Coronavirus: Young Canadians with rare diseases feeling impact of pandemic
March 16, 2021

“It’s bittersweet because he’s taking a big brother role, even though he’s the little brother,”...