Hope
needs
help.
My name is Dr. Eric Hoffman, and I am a human geneticist and translational researcher focused on neuromuscular disease. I am writing to you alongside my esteemed colleague, Dr. Jean Mah, who is the Director of the Pediatric Neuromuscular Program at the Alberta Children’s Hospital in Calgary.
About ten years ago, we began a project to understand the limitations of corticosteroids, the current and only treatment available for Duchenne muscular dystrophy in Canada and to see if alternatives might be developed.
We have spent decades exploring new and improved ways to extend and enhance the quality of life for children and young adults coping with this debilitating disease.
Steroids have been the most frequently recommended drug for boys experiencing the loss of muscle, mobility and independence associated with Duchenne.
They serve to slow and mitigate some of these symptoms, but they come with a long list of dangerous side effects, including:
- Stunted growth
- Brittle bones
- Mood disturbances
- Profound impact on the
hormonal health of young men
Our research led to the development of a possible safer alternative called vamorolone.
By changing the mechanism of corticosteroids, we have created a medication that provides Duchenne patients with improvements in muscle strength and function while mitigating some of the risks typically associated with steroidal treatments.
We carried out a series of clinical trials in boys 4 to 7 years old that have been promising; however, a lack of funding prevented our team from opening Canadian clinical trials for older and younger children.
This is where you have made an enormous difference.
In 2020, your generosity made a $750,000 investment in our research possible. This grant allows us to test vamorlone with a broader age range, resulting in greater patient access and involvement in clinical trials.
Thanks to you and our long-time partners at Defeat Duchenne Canada, we are countless steps closer to bringing our novel vamorolone treatment to families coast to coast.
While our team gets excited about the scientific breakthroughs, what matters most are the human benefits of the research your helped fund. We are honoured to share this journey with the Masnada family, who explains what it is like to participate in a vamorlone clinical trial:
Vamorolone has given Ollie strength and energy, two things he was already running low on.
By getting involved in this research trial, we feel like we are giving Ollie more time, until the real cure comes hopefully one day. I can’t tell you how much peace that gives us, in knowing we are doing all we can to support and save our son.
– Nikol Masnada (Ollie’s Mom)
In addition to getting the benefits of stronger, more able bodies, the Defeat Duchenne Canada-supported clinical trial of vamorolone may mean we can begin treating kids sooner. Rather than waiting three, four, or five years for answers and support, early screening may set kids up for success even before they can crawl. Through further research, we can passionately pursue a future where a life with Duchenne is radically different than what has become considered the status quo.
With your support, we can help stabilize this disease, extend independence and change the course of Duchenne muscular dystrophy.
This progress is exciting – for us, the families we serve, and the healthcare partners eager to save and improve lives. But we cannot maintain this momentum without you. When you choose to donate, you are bridging the gap between what is explored in a lab and what is making a real difference to those living with Duchenne, like Ollie.
Invest in the bravery of patient families, like the Masnada’s, who put themselves through the challenges of participating in clinical trials for the idea that tomorrow may be brighter for their child and children around the world.
Please donate to enable researchers - like us - to continue making ground-breaking discoveries and improve care.
Thank you,
Dr. Eric Hoffman
Dr. Jean Mah
Research is the road to hope, but hope needs help.
With your support, we can help stabilize this disease, extend independence and change the course of Duchenne muscular dystrophy for children, like Ollie.