July 2021 Newsletter

The July 2021 issue of the NMD4C newsletter is now available!

We share a new publication Guidance on gene replacement therapy in Spinal Muscular Atrophy: a Canadian perspective, and launch the new clinical trial concierge web page. We also share news about a new EPIC functionality helping to keep myotonic dystrophy patients safe at The Ottawa Hospital, the ENMC's new Themed workshops, Knowledge Translation updates, a 2021 Walk4MD wrap-up & more!

View the July 2021 newsletter here.

Sign up for future issues of the NMD4C newsletter here.

New Publication Guidance on gene replacement therapy in Spinal Muscular Atrophy: a Canadian perspective

Myotonic Dystrophy: EPIC Functions at The Ottawa Hospital

July Knowledge Translation Update

European Neuromuscular Centre Themed Workshops

Clinical Trial Concierge Web Page Launch

2021 Walk4MD Wrap-Up

Join  the NMD4C

New Research from our Members

Guidance on gene replacement therapy in Spinal Muscular Atrophy: a Canadian perspective

  • Dr. Maryam Oskoui, with Drs. Hernan Gonorazky, Hugh McMillan, James Dowling, Reshma Amin, Cynthia Gagnon, and Kathryn Selby as co-authors.

 

A Randomized, Double-Blind, Placebo-Controlled, Global Phase 3 Study of Edasalonexent in Pediatric Patients with Duchenne Muscular Dystrophy: Results of the PolarisDMD Trial

  • Dr. Richard Finkel, with Dr. Hugh McMillan (investigator) as a co-author.

 

Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy

  • Dr. Elizabeth Kichula with Dr. Hugh McMillan (Investigator) as a co-author.

 

Biomarkers in Duchenne and Becker muscular dystrophies.

  • Dr. Hugh McMillan (investigator) with Dr. Hanns Lochmüller (lead investigator) as a co-author.

 

Metabolic Dysfunction in Spinal Muscular Atrophy

  • Dr. Marc-Olivier Deguise, with Dr. Rashmi Kothary (steering committee) as a co-author.