Medical mystery: A teenager’s apparent clumsiness foreshadowed a shocking diagnosis

Judy Kalnas remembers thinking that Jessica, the youngest of her six children, resembled the elastic-limbed...

GM2 Gangliosidosis Research (Sandhoff and Tay-Sachs)

GM2 Gangliosidosis (Sandhoff and Tay-Sachs) Research Overview Sandhoff is a lysosomal storage disorder. It is...

Tay Sachs: Victorian mum with two terminally ill children tells her story

Kelly McElligott and her family. Credit: Supplied Kelly McElligott, from Victoria, is living every mother’s...

UMass Med School gene therapy shows promising early results in tackling Tay-Sachs

UMass Medical School Dean Dr. Terence R. Flotte is interviewed in his office about a...

Promising Results Reported in Tay-Sachs Gene Therapy Trial

BARCELONA – Researchers have obtained the first signs of clinical benefit in an early-stage gene...

Axovant Presents First Evidence of Clinical Stabilization in Tay-Sachs Disease at the European Society of Gene and Cell Therapy 27th Annual Congress

Initial data with AXO-AAV-GM2 suggests stabilization of disease course, attainment of normal developmental milestones, and...

Genome editing with precision

Prime editing system offers wide range of versatility in human cells, correcting disease-causing genetic variations...

$28 million grant supports Cincinnati Children’s role as coordinating center for Rare Diseases Clinical Research Network

The federal agency that oversees a large network of research centers seeking cures for hundreds...

New hope for Tay-Sachs disease at UMass Medical School

LISTEN Produced by UMass Medical School · New hope for Tay-Sachs disease A new Voices...

AXOVANT announces clinical update from first Tay-Sachs disease patient dosed with AXO-AAV-GM2 gene therapy

Generally well-tolerated with no serious adverse events in a 30-month-old patient with advanced infantile Tay-Sachs...