News – Page 15 – Muscular Dystrophy Canada

Federal Government Commits to National Strategy to Improve Access to Rare Disease Drugs

Toronto, Ontario – Muscular Dystrophy Canada commends the Government of Canada for committing $35 million, in its 2019 budget, to develop a Canadian Drug Agency to oversee the development of a new national formulary of prescribed drugs and a strategy to provide support for Canadians with rare diseases.

A national strategy to alleviate the high-cost of drugs for rare diseases will ensure more Canadians are able to access effective, life-changing treatments.

“This is an important first-step towards ensuring individuals diagnosed with a rare disease, such as a neuromuscular disorder, are able to access treatment without cost as a barrier,” said Barbara Stead-Coyle, CEO, Muscular Dystrophy Canada. “We call on all Federal parties to focus on this very real issue facing Canadians.”

Rachel Andrews Story

It’s been a little over a year since I sat in a Neurologists office, with my husband by my side, and heard the words “I believe you have Facialscapulohumeral Dystrophy (FSHD)”.

My first response was, “say what?” It’s a mouthful for sure, but I’m fortunate my failing facial muscles are still able to say it.

Hello, my name is Rachel and I wanted to share my story with you because Muscular Dystrophy has impacted my life since well before my diagnosis. And, I believe more research and education will ensure Canadians like me don’t continue suffering with undiagnosed symptoms.

CADTH REVIEW AND SPINRAZA

Spinraza decision signals the need for a Canadian Rare Disease (or Orphan Drug) Framework

The drug review and approval process in Canada is complex. Many agencies from Health Canada, to Canadian Agency for Drugs and Technologies in Health (CADTH) and Institut national d’excellence en santé et en services sociaux (INESSS), to the Pan-Canadian Pharmaceutical Alliance and each individual provincial and territorial governments all share in the decision-making.

MDC Welcomes Daria Wojtal as Director of Research

We are pleased to announce that Daria Wojtal has joined Muscular Dystrophy Canada as the new Director of Research. Daria comes to us from the Hospital for Sick Children and the University of Toronto, where she worked under the leadership of Dr. Ronald Cohn on her PhD in developing CRISPR gene editing technology for the treatment of neuromuscular disorders.