Canada’s Drug Agency, in collaboration with the Institute for Clinical Evaluative Sciences (ICES) and the Health Data Research Network Canada (HDRN), has released a roadmap to help patient and disease registry teams link data with health services records, advancing rare disease research and care.

A recent study published in Science has identified crucial genetic mutations in the TRiC chaperonin complex linked to severe brain malformations, offering new diagnostic and treatment possibilities for rare disease patients worldwide, with 22 individuals diagnosed so far through international collaboration led by Dr. Hanns Lochmüller.

The ALS Society of Canada (ALS Canada) celebrates the Ontario government’s $13 million investment over three years to support the Provincial ALS Program. This program will expand access to multidisciplinary care, assistive devices, and essential services.

Led by Dr. Carl Ernst and a collaborative team from The Neuro, Montreal General, Montreal Children’s Hospital, and McGill University, the project will compare various gene therapy delivery methods for safety and effectiveness to overcome existing treatment barriers.