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. The network is incredibly fortunate to be able to collaborate with these incredible organizations, and it is our pleasure to recognize the contributions of each towards helping to fund neuromuscular research.
Jesse’s Journey
Jesse’s Journey, Canada’s leader in Duchenne muscular dystrophy funded research, will grant $1.7M toward four promising research projects this year – the largest annual amount in its 26-year history.
Newly funded projects in 2021 include:
Dr. Niclas Bengtsson: Development of gene regulatory cassettes that enable safe- and efficient in vivo dystrophin gene-editing in muscle stem cells.
Dr. Natasha Chang: Inducing stress granule formation in muscle stem cells to treat DMD.
Dr. Ronald Cohn: Simultaneous DMD gene editing and upregulation to restore protective levels of full-length dystrophin in the treatment of DMD duplication mutations.
Dr. Sachiko Sato: Development of a Monosaccharide Therapy Using N-Acetylglucosamine to Mitigate Duchenne Muscular Dystrophy.
The growth in scientific funding interest is encouraging for the community because it means there is an increasing commitment to Duchenne science and drug discovery, bringing hope and progress towards prevention, treatment, and ultimately – a cure.
Read the full press release from Jesse’s Journey here!
Muscular Dystrophy Canada
Muscular Dystrophy Canada (MDC) has announced an investment of $400,000 to fund seven new research projects in 2021 through the MDC Neuromuscular Disorder (NMD) Research Grant Competition, a dedicated Canadian source of funding for neuromuscular research. MDC will also invest in an international, collaborative, $1.15 million, research project through the European Joint Programme on Rare Diseases (EJP RD).
The 2020-2021 research grant recipients are:
Dr. Alex Parker: A Pharmacogenetic Pipeline for Charcot-Marie-Tooth Disease
Dr. Colin Crist: Promoting Muscle Repair by Pharmacological Inhibition of eIF2a Dephosphorylation
Dr. Mohamed Chahine: Human iPSC-derived Neurons as a Model of Congenital Myotonic Dystrophy Type 1
Dr. Nadine Wiper-Bergeron: Improving Myoblast Transplantation Outcomes via Pharmacological Reprogramming
Dr. Nicolas Dumont: Targeting Defective Stem Cells in a Preclinical Model of Myotonic Dystrophy Type 1
Dr. Rima Al-Awar (EJPRD/CIHR Co-funded): Safety and Efficacy of a Possible Epigenetic Therapy for Facioscapulohumeral Muscular Dystrophy
Dr. Toshifumi Yokota: Enhancing the Efficacy of Antisense Oligonucleotide Therapy for Facioscapulohumeral Muscular Dystrophy
Read the full press release from Muscular Dystrophy Canada here!
Read next...
New Publication from the NMD4C Clinical Trial Network Strengthens Trial Capacity in Canada
We are pleased to share a new publication from the NMD4C Clinical Trial Network led by Kerri Lynn Schellenberg, Homira Osman, Maria Masnata, Rhiannon Hicks, Corinne Kagan, Ana Stosic, Stacey Lintern, Erin Beattie, Drs. Hanns Lochmüller, Craig Campbell, and Jean K Mah. This work focuses on the implementation of a coordinated Canadian neuromuscular clinical trial network to enhance trial readiness, capacity, and access in rare disease research.
Canadian-Led Phase II Studies Advance Vamorolone Evidence in Duchenne Muscular Dystrophy
NMD4C investigators contributed to two phase II studies evaluating vamorolone in boys with Duchenne muscular dystrophy across early childhood and adolescence, demonstrating safety, favorable pharmacokinetics, and potential efficacy. Led by Dr. Jean Mah and conducted across Canadian clinical trial sites with national and industry collaborators, the findings supported Health Canada approval of vamorolone as the first treatment for DMD in Canada. Leaders from both the clinical and patient advocacy communities, including Defeat Duchenne Canada, highlighted the impact of collaboration among patients, families, clinicians, and researchers in advancing care and improving access to new therapies.
Advancing AFC in Neuromuscular Medicine: Program Launch & September Workshop
The Royal College has approved National Training Standards for the AFC in Neuromuscular Medicine, creating a formal pathway to recognize enhanced competence with the DRCPSC credential. This designation establishes a national training standard, supports portable certification, and strengthens clinical capacity across Canada. PGME offices can now apply to offer accredited AFC programs, with a goal of five programs submitting applications by the end of 2026.