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...

SickKids researchers ‘model’ future of precision medicine for rare inherited disorders

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

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

Sio Gene Therapies Announces First Patient Dosed in Clinical Trial of AXO-AAV-GM2 in Patients with Tay-Sachs and Sandhoff Disease (GM2 Gangliosidosis)

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Feb. 03, 2021 (GLOBE NEWSWIRE) — Sio Gene...