The Neuromuscular Disease Network for Canada


Exciting research grants awarded by Jesse’s Journey

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.


Read next...


October 2020 newsletter

The October 2020 issue of the NMD4C newsletter is now available!

Topics include a 10-year reflection of the Canadian Neuromuscular Disease Registry, our process for selecting clinical guidelines to adapt, our booth at the Myotonic Dystrophy Foundation virtual conference, recent and upcoming webinars, new research from NMD4C participants, and a member spotlight on steering committee member Stacey Lintern.

About us

We’re hiring a communications coordinator!

We are looking for a full-time communications coordinator for a term position ending March 2022. This position is located at the NMD4C coordination office at the CHEO Research Institute in Ottawa, Ontario, but will be working from home until at least June 2021.


NMD4C investigator Bernard Brais to be awarded The Norman Saunders Jacob’s Ladder International Research Prize

We are very proud of our investigator Dr. Bernard Brais for receiving the 2020 Norman Saunders Jacob’s Ladder International Research Prize! Dr. Brais will be giving a talk at his award presentation on October 7, 2020, entitled “Ataxias with regional founder effects in Québec: Lessons on diversity.” 


Latest edition of the Journal of Neuromuscular Diseases now online – Sep 2020

A new issue of the Journal of Neuromuscular Diseases has recently been released: Volume 7, Issue 4 (Sep 2020)


CME-accredited webinar | Interdisciplinary guidelines for myotonic dystrophy type 1 (DM1) management across the spectrum

NMD4C and MDC are pleased to invite you to a CME-accredited webinar on interdisciplinary guidelines for myotonic dystrophy type 1 (DM1).


NMD4C steering committee member Stacey Lintern appointed CEO of Muscular Dystrophy Canada

We are happy to congratulate our steering committee member Stacey Lintern for her recent appointment as CEO of our partner organization Muscular Dystrophy Canada (MDC)!