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...

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

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

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...

Taysha Gene Therapies Announces Queen’s University’s Receipt of Clinical Trial Application Approval from Health Canada for Phase 1/2 Clinical Trial of TSHA-101 for the Treatment of Infantile GM2 Gangliosidosis

DALLAS–(BUSINESS WIRE)–Dec. 21, 2020– Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a patient-centric gene therapy company...

Health Canada approves $2.8M treatment for spinal muscular atrophy

Health Canada has approved Zolgensma, which is bringing renewed hope to Alberta families that have...

Sio Gene Therapies Announces Positive Six-Month Follow-Up Data from Low-Dose Cohort of Phase 1/2 Trial of AXO-AAV-GM1 for GM1 Gangliosidosis

NEW YORK and RESEARCH TRIANGLE PARK, N.C., Dec. 15, 2020 (GLOBE NEWSWIRE) — Sio Gene...