The Neuromuscular Disease Network for Canada

funder-logos_nmd4c

A national spinal muscular atrophy registry for real world evidence

One of NMD4C’s many important initiatives is the development of neuromuscular disease (NMD) patient registries. Patient registries collect information about individuals who are affected by a particular condition and are a particularly important research and networking tool for rare diseases. The data collected may be used for a range of purposes, from research into specific features of the disease to clinical trial feasibility planning and recruitment. Patients who join registries can be contacted with information relevant to their condition and notified when they may be eligible for research studies and clinical trials.

NMD4C works closely with the Canadian Neuromuscular Disease Registry (CNDR), a Canada-wide registry of people diagnosed with an NMD. It collects important medical information from patients across the country to improve the understanding of NMD and accelerate the development of new therapies. Over 4500 NMD patients have registered from across Canada since the CNDR’s launch in 2010. With their data, the CNDR has facilitated 35 trials and 75 data inquiries and research projects.

Recently, the CNDR undertook an iterative multi-stakeholder process to expand its spinal muscular atrophy (SMA) dataset to capture items relevant to patient outcomes in the use of the new therapies such as nusinursen (Spinraza). This will serve as a longitudinal, prospective, multi-centre, observational study of patients with SMA in Canada designed to evaluate the safety and effectiveness of novel therapies and provide practical information unattainable in trials. In the development of this new dataset, the team also aimed to standardize outcome measures across the network and the broader Canadian community, and to align its measures with global datasets to facilitate collaboration. The data collected with this expanded dataset will be essential to inform improvements in care and access to therapy for all SMA patients.

Click here to read more about this project

Many NMD4C investigators and members were on the team involved in expanding the CNDR’s SMA dataset:

NMD4C steering committee involved in the project:

NMD4C investigators involved in the project:

NMD4C collaborators involved in the project:

NMD4C members involved in the project:

  • Nicolas Chrestian
  • Susan Dojeiji
  • Nicolas Dupré
  • Hernan Gonorazky
  • Aaron Izenberg
  • Alier Marerro
  • Anna McCormick
  • Gerald Pfeffer
  • Stephanie Plamondon
  • Xavier Rodrigue
  • Jordan Sheriko
Annotation 2020-07-03 092543

Read next...

Diagnostic-Techniques-Genetic-Neuromuscular-Diseases-Register-Now-1200x1200

CPD-accredited webinar | Diagnostic techniques for genetic neuromuscular diseases

NMD4C and MDC are pleased to invite you to a CPD-accredited webinar on diagnostic techniques for genetic neuromuscular diseases.

email-3249062_1280

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.

bernard-brais

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.” 

JND-cover

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)

Clinical-Guidelines-Register-Now-1200x1200

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).