The Neuromuscular Disease Network for Canada

One of NMD4C’s partners, Jesse’s Journey (a Canadian charity dedicated to funding research on Duchenne muscular dystrophy), recently announced their research grant recipients for 2020.

NMD4C is very excited that two of our investigators were awarded funding!

Dr. Lawrence Korngut is a neurologist and clinical neurophysiologist. Dr. Korngut has been instrumental in the creation of the Canadian Neuromuscular Disease Registry (CNDR), a Canada-wide registry of people diagnosed with a neuromuscular disease. It collects important medical information from patients across the country to improve the understanding of neuromuscular diseases and accelerate the development of new therapies. Currently, over 4500 neuromuscular patients have registered from across Canada. Jesse’s Journey is one of the original funders of the CNDR and will be funding it for its 12th consecutive year.

Dr. Craig Campbell is a pediatric neurologist. Dr. Campbell is the Head of pediatric neurology at the London Health Sciences Centre Children’s Hospital, which is a lead site for myoblast transplantation in boys with Duchenne. Jesse’s Journey will continue its previous funding of this project into 2020. The project is currently under review; stay tuned for further details!

Another of Jesse’s Journey’s research grants for 2020 was exciting for a different reason — it was the first time they awarded $1 million to a single project!

The project is a Canadian clinical trial evaluating vamorolone in children 2-4 years of age prior to the onset of Duchenne, and in adolescents 7-18 years of age already living with Duchenne — an age range often neglected in clinical trials.

Dr. Eric Hoffman and his team at ReveraGen BioPharma have found a way to tweak the chemistry of standard corticosteroids, the current standard of care, and developed a new molecule that retains the anti-inflammatory benefits but loses much of the activity associated with side effects. ReveraGen has successfully completed Phase I and II programs demonstrating that treatment with vamorolone led to improvements of strength and mobility over six months and preserved over 1.5 years of treatment. Importantly, key side effects, such as stunting of growth, were not observed, and the boys grew normally. This data is supportive of vamorolone having the potential to replace corticosteroids as the standard of care treatment for children and young adults with Duchenne.

Read more about this clinical trial HERE.

See more research funded by Jesse’s Journey HERE.