Axovant Gene Therapies Announces FDA Clearance of IND for AXO-AAV-GM2 Gene Therapy in Tay-Sachs and Sandhoff Diseases

AXO-AAV-GM2 is the first investigational gene therapy to receive IND clearance in Tay-Sachs and Sandhoff...

Nobel Prize in Chemistry 2020: Genetic scissors: a tool for rewriting the code of life

Emmanuelle Charpentier and Jennifer Doudna are awarded the Nobel Prize in Chemistry 2020 for discovering...

Axovant and Viralgen Sign Strategic Gene Therapy Development and Manufacturing Partnership

Partnership secures access to cGMP capacity and resources at Viralgen to support the development and...

A six-month feasibility study evaluated patients’ ongoing compliance in wearing digital health technology in order to capture continuous data remotely as well as changes often missed in between hospital visits.

Abstract Background As part of a late onset GM2 gangliosidosis natural history study, digital health...

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...

Together in Worship. The Music of Lent

Friday, February 21, 2020 at 7:30pm...

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...