Health – Muscular Dystrophy Canada

FUNDING FOR INNOVATIVE THERAPIES FOR HEREDITARY ATAXIAS

For immediate release

Montréal, April 28, 2023 – It is with great enthusiasm that Génome Québec, Ataxia Canada and Muscular Dystrophy Canada announce the launch of the Innovative Therapies for Hereditary Ataxias competition to encourage the discovery and development of new therapies and to mobilize the research ecosystem. The three organizations aim to jointly invest $1M to support projects with a maximum of $330,000 per project for a period of two years.

The goal of this competition is to meet the needs of people affected by these rare neurological diseases, to enable them to benefit from genomic innovations and to encourage the development of innovative therapies. It supports the objectives and orientations of the provincial and federal governments, notably by accelerating therapeutic innovation (Politique québécoise pour les maladies rares) and by supporting research to improve access to affordable and effective drugs for the treatment of rare diseases.

This competition is open to researchers affiliated with a Québec university and its affiliated institutions (including hospitals and research institutes).

The deadline for submitting the mandatory registration to Génome Québec is June 8, 2023.

Learn more “FUNDING FOR INNOVATIVE THERAPIES FOR HEREDITARY ATAXIAS”

Funding for first-ever National Strategy for Drugs for Rare Diseases announced

FOR IMMEDIATE RELEASE March 22, 2023

Toronto, Ontario – Muscular Dystrophy Canada applauds the Government of Canada for committing $1.5 billion over three years in support of a National Strategy for Drugs for Rare Diseases to help increase access to, and affordability of, promising and effective drugs for rare diseases to improve the health of patients across Canada.

“MDC works with Canadian Fire Fighters, donors, volunteers and other like-minded organizations to break down barriers for the neuromuscular community. The major barriers that exist for our diverse community, which consists of individuals with rare and ‘ultra-rare’ neuromuscular disorders, are lack of cures and delayed – out of reach treatments. So this announcement is great news for our community,” said Stacey Lintern, CEO, Muscular Dystrophy Canada. She added, “we are excited that this announcement has been made but time is of the essence for many rare diseases and the sooner we can move this from strategy to action, the better it is for our community.”

This long-awaited announcement identified four areas of focus for the national strategy and funding support for provinces and territories to improve access to new and emerging drugs, enhance access to existing drugs, early diagnosis, and screening for rare diseases.

“The areas of focus align perfectly with the work that MDC is already doing such as ensuring early diagnosis and clinical genetic testing. We know access to an accurate diagnosis, coupled with early access to treatments, are important for optimal outcomes and better quality of life,” said Lintern.

“MDC welcomes a strategy where the patient and family are at the forefront and that supports an evidence-based care journey. We are committed to working with our partners like the Neuromuscular Disease Network for Canada (NMD4C) to help make that happen, and bring about better diagnostic tools, support for clinical trials and evidence that supports decision-making. We look forward to a time when the neuromuscular community has access to accurate and early diagnoses, life-changing treatments, and supports regardless of where they live, their age or the rarity of the condition.”

The full Government of Canada announcement can be read here

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ABOUT MUSCULAR DYSTROPHY CANADA

Muscular Dystrophy Canada’s mission is to enhance the lives of persons affected by neuromuscular disorders by continually working to provide ongoing support and resources while relentlessly searching for cures through well-funded research. To learn more about MDC, please visit muscle.ca or call our toll-free number at 1-800-567-2873.

FOR MORE INFORMATION CONTACT:

Heather Rice
Muscular Dystrophy Canada
Heather.Rice@muscle.ca
902-440-3714

B.C. newborn screening expands; early detection improves quality of life

Muscular Dystrophy Canada is overjoyed that babies born in British Columbia will now be screened for spinal muscular atrophy (SMA). Congratulations to the Government of B.C. on taking this important step that will lead to early diagnosis and treatments that will have life-changing results for individuals and families. Access to the earliest diagnosis will ensure that patients have access to the right treatment and healthcare at the right time, resulting in the best health outcomes for patients.

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.”

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.