The Neuromuscular Disease Network for Canada

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Congenital Myotonic Dystrophy Trial Now Recruiting

The NMD4C is excited to share that an international randomized, multicenter, double-blind, placebo-controlled, phase 2/3 clinical trial testing the safety and efficacy of Tideglusib in Congenital Myotonic Dystrophy (CDM) patients aged 6-16 has begun recruiting. This is the first CDM trial to take place in Canada, even though CDM is one of the more common neuromuscular diseases in Canada, affecting more than one in 50,000 live births. The two Canadian sites are the Children’s Hospital London Health Sciences Centre (LHSC) in London, ON and the Children’s Hospital of Eastern Ontario (CHEO) in Ottawa, ON, with Drs. Craig Campbell and Hanns Lochmüller as Principal Investigators.

Both the London and Ottawa sites are open for recruitment; contact information for both sites is located below. If you would like to learn more about the eligibility criteria and for further information, please visit the ClinicalTrials.gov site to find detailed information!

LHSC site: Rhiannon Hicks, , (519)-685-8441.

CHEO site: Emilie Hill-Smith, , 613-737-7600 ext. 4014.

Our network brings together clinicians, researchers, and people with neuromuscular disease across the country.

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JND treatabolome

Treatabolome Project Designed to Shorten Diagnosis-to-Treatment Time for Patients with Rare Diseases

The NMD4C would like to share the publication of a special issue of the Journal of Neuromuscular Disease dedicated to the Treatabolome, a new online database for rare disease and gene-specific treatment information. The special issue includes six systematic reviews contributed by experts on rare neurological disorders.

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Neuromuscular Research Funding Announcements From Jesse’s Journey and Muscular Dystrophy Canada

The NMD4C is excited to share recent grant funding announcements from two of our partners, Jesse’s Journey and Muscular Dystrophy Canada. Jesse’s Journey will grant $1.7M toward four promising research projects this year. Muscular Dystrophy Canada (MDC) will invest $400,000 towards seven new research projects through the MDC Neuromuscular Disorder (NMD) Research Grant Competition and through the European Joint Programme on Rare Diseases (EJP RD).

newborn screeningMay

Muscular Dystrophy Canada Announce Phase 2 of Newborn Screening Initiatives with Call for Funding Proposals

MDC is excited to launch Phase 2 of their work with a call for proposals for local newborn screening for SMA initiatives. It is anticipated that these projects will be led by labs or clinicians (or both) and should either focus on setting the lab up for testing or ensuring efficient uptake of policy (i.e., addition of SMA to existing provincial newborn screening panel).

EC Update

Early Career Working Group Update

the NDM4C has created a list of profiles for these members, where you can learn more about their research interests, publications, and where you can find them online. The NMD4C has also launched an early-career Slack channel, and are seeking early career member representation on our work packages.

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Read Dr. Louise Moyle’s Early Career Blog

Read the latest early career blog post, from NMD4C member Dr. Louise Moyle. 

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Jesse’s Journey Grants $1.7 Million to Duchenne Muscular Dystrophy Research

Jesse’s Journey, Canada’s leader in Duchenne muscular dystrophy funded research, will grant $1.7M toward four promising research projects this year.