Susi Vander Wyk
Executive Director, Cure SMA Canada
Spinal muscular atrophy is a complex rare disease that affects approximately one in 6,000 babies born. Without treatment, it’s possible that children won’t be able to meet important developmental milestones like sitting up on their own or walking. Because of this, access to treatment is extremely important.
We sat down with Susi Vander Wyk, the Executive Director of Cure SMA Canada, to understand how research and development could help increase access to treatment for Canadians living with rare diseases.
What does investment in Canadian research and development in rare diseases mean to you?
Investing in rare disease research and development provides hope for a future for patients affected by rare diseases. Many of these diseases are progressive and debilitating by nature. Without the development and ultimate access to these therapies, the future is frightening for what it holds. Treatment changes that fear to hope and planning for tomorrow.
When talking about the future of gene therapies, what’s the outlook from a patient perspective?
Innovative treatments such as gene therapy completely change the course of the disease. We have patients who were fortunate to access gene therapy through clinical trials or a managed access program who experienced meeting physical milestones that were absolutely unreachable to patients without access in the past. These families will never have to experience the devastation of helplessly watching their children grow weaker and weaker, and they’ll never have to come to terms with the impact of the fear of illness and death of a member of their family.