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February 2025 Newsletter

February 2026 NMD4C Newsletter
   

News

   

2026 Collaborative Research Training Awards

We are excited to share that the second cycle of our Collaborative Research Training Award program is now open for applications! 
The NMD4C Collaborative Research Training Award provides $2,000 for research trainees to visit another neuromuscular lab within Canada. This opportunity enables trainees to work on a collaborative research project while gaining hands-on experience, learning new techniques, and further developing their expertise in neuromuscular disease focused research. We look forward to supporting the innovative research and professional growth of our trainees through this exciting program. You can learn more about the application process and guidelines on our website. 

Applications will be accepted until June 15, 2026.

Save the Date – 2026 NMD4C Annual Meeting

Our annual meeting will be held in Calgary on Friday, September 25, 2026Join us for a full day of dynamic discussions, research updates, and networking opportunities, just ahead of the 6th Western Canadian Neuromuscular Conference (WCNMC 2026).

This year, we’re excited to share a special opportunity for research trainees; For the first time, WCNMC 2026 will host a full day of basic science sessions, running in parallel with the clinical sessions on Saturday September 26, followed by a networking event. All basic science trainees who attend the NMD4C Annual Meeting will receive complimentary registration for the WCNMC 2026 basic science sessions and networking event!

Whether you’re a clinician shaping future guidelines or a researcher seeking interdisciplinary collaboration, the 2026 NMD4C Annual Meeting promises meaningful discussion and actionable insights.

More Details, Coming Soon! 

Clinical Trial Network: Mentorship Opportunity

Our Neuromuscular Clinical Trial Network is committed to empowering junior investigators and neuromuscular specialists who are interested in leading clinical trials. We offer personalized, one-to-one mentorship designed to build the skills, confidence, and practical expertise required for successful trial leadership and conduct. Participants can be matched with experienced trialists who bring 10+ years of experience leading high-impact clinical trials across a range of neuromuscular diseases. 
Mentorship pairings are tailored to your goals and interests, with matching based on therapeutic area, pediatric or adult populations, geographic location, team size, and other key considerations. Reach out to our Clinical Trial Network Manager, Maria Masnata to learn how we can support your development as a clinical trial leader.

Congratulations to NMD4C Investigators Dr. James Dowling and Dr. Laura McAdam on Receiving 
2025 Defeat Duchenne Canada Research Grants!

We are happy to share and congratulate our NMD4C Investigators,
Dr. James Dowling and Dr. Laura McAdam on being awarded grant funding from Defeat Duchenne CanadaWe are excited to see how these investigators continue to advance treatments, strengthening supports, and improving quality of life for people living with Duchenne.

Dr James Dowling,
University of Pennsylvania

Project:

Non-Viral Gene Therapy for Duchenne Muscular Dystrophy

Dr Laura McAdam,

Holland Bloorview Kids Rehabilitation Hospital. 

Project:Youth Helping Youth: Dissemination of a User Informed Bullying Resource for Youth with DMD

     
     
   
   

CPD-Accredited Webinars 

NMD4C and Muscular Dystrophy Canada are pleased to invite you to our next CPD‑accredited webinars on the Charcot Marie Tooth Disease: Clinical and Research Updates and Duchenne Muscular Dystrophy in the New Steroid Age: Practical Considerations

These sessions are designed for clinicians, researchers, and trainees with an interest in neuromuscular diseases.

Charcot Marie Tooth Disease: Clinical and Research Updates

Details: 

  • Date: Tuesday, Feb 17, 2026 
  • Time: 5-6 pm ET

Chair: Dr. Alberto Aleman

Speakers: 

  • Dr. Rami Massie: Update on CMT1A. Clinical trials and genetic therapies. 
  • Dr. Rita Horvath: Peripheral neuropathy in mitochondrial diseases 

Duchenne Muscular Dystrophy in the New Steroid Age: Practical Considerations

Details: 

  • Date: Tuesday, April 7, 2026 
  • Time: 5-6 pm ET

Chair: Dr. Leanne Ward

Speakers: 

  • Dr. Anne Marie Sbrocchi:
    Duchenne Muscular Dystrophy in the Evolving Steroid Era: Practical Neuromuscular and Endocrine Considerations
  • Dr. Jean Mah: Vamorolone: A novel treatment for Duchenne muscular dystrophy (DMD)

ERN Euro-NMD Webinars:
Genetics Month Series

As part of Genetics Month, EURO-NMD is pleased to host a four-episode webinar series bringing together clinicians, researchers and experts involved in genetics and rare neuromuscular diseases. 
Throughout February 2026, every Thursday, these webinars will provide an opportunity to share experiences, discuss current practices and explore how genetics is shaping diagnosis, care pathways, emerging therapies, and the impact of newborn screening on rare diseases. Each session combines scientific insight with practical perspectives, fostering knowledge exchange across the European research and clinical community.

Episode 1: ERDERA Diagnostic Research Workstream

This webinar was held on, 5 February 2026, watch the replay!

Episode 2: CPMS Use and Value for Genetic Case Discussions

Date: Thursday, 12 February 2026

TIme: 16:00 CET/ 10:00 AM EST

Episode 3: Genetics of Amyotrophic Lateral Sclerosis and Impact on New Therapies

Date: Thursday, 19 February 2026

TIme: 16:00 CET/ 10:00 AM EST

Episode 4: 

Newborn Screening and Impact on Rare Diseases

Date: Thursday, 26 February 2026

TIme: 16:00 CET/ 10:00 AM EST

Stem Cell Network: Careers Beyond Academia

Stem Cell Network and Medicine by Design are hosting a virtual seminar series to help trainees understand the different careers available outside of academia, the skills required for certain positions, and some of the daily functions of those positions.

  • February 18, 2026: Clinical Research 
  • March 18, 2026: Entrepreneurship 

George Karpati Lecture: The Era of Bespoke Therapies 

for Genetic Neuromuscular Diseases

The Annual George Karpati Lecture honours a world-renowned neurologist and clinician-scientist who dedicated his remarkable career to The Neuro. Dr. Karpati was celebrated for advancing the diagnosis, treatment, and understanding of neuromuscular diseases, especially muscular dystrophy. Each year, his colleagues host this lecture to highlight recent progress in neuromuscular research and care, paying tribute to a beloved friend, mentor, and scientific leader.

Date: Tuesday, February 24, 2026  

Time: 16:00 to 18:00 ET 

World Muscle Society: Myology Café 

Join the global myology community for the next online Myology Café on Thursday, 26 February 2026 at 8:00 AM EST (13:00 GMT). This session launches a new series exploring regional approaches to NMD care and patient registries. Hosted by Juliana Gurgel‑Giannetti, it will feature Gisela Nogales presenting insights from the Registry of Myotonic Dystrophy 1. 

     
   

New Publications & Research

   

See a list of January 2026 publications from NMD4C members on PubMed! 

     
     
   

Community News 

   

Defeat Duchenne Canada: Celebrating 30 Years of Advancing Duchenne Research

On January 30, Defeat Duchenne Canada announced this year’s Research Grant recipients and the funding being dedicated to innovative projects focused on improving the lives of individuals living with Duchenne muscular dystrophy. The announcement also marked a major milestone: surpassing $20 million invested in Duchenne research since 1995

This progress reflects 30 years of collaboration, community, and belief in research as the pathway to better treatments, care, and outcomes for families affected by Duchenne. Learn more about the newly funded projects and this milestone achievement:

     

15th International Myotonic Dystrophy Consortium Meeting (IDMC-15) – Registration is Open!

The IDMC-15 Organizing Committee would like to inform you that registration is open! The event will offer an incredible opportunity to learn about recent advancements, exchange ideas, and build connections among all those involved in the field of myotonic dystrophy: researchers, clinicians, patients, patients partners, patient associations, and representatives and professionals from the pharmaceutical industry. 
  • Date: May 26-30, 2026 
  • Location: Hôtel Le Montagnais, Chicoutimi, Québec

     

WCNMC 2026: Early Registration & Call for Abstracts

Registration is now open for the 6th Annual Western Canadian Neuromuscular Conference (WCNMC)! Clinicians, researchers, trainees, and allied health professionals are encouraged to submit abstracts.

  • Oral presentations - March 1st, 2026: Individuals selected for oral presentations will receive registration reimbursement and support for travel/accommodation.
  • Poster presentations - September 1st, 2026: Trainees presenting accepted posters will receive reimbursement of registration after the conference.

* Basic science trainees attending the 2026 NMD4C Annual Meeting will receive complimentary registration to the WCNMC, making this an excellent opportunity to present research and engage with the broader neuromuscular community. 
* Super Early Bird rates offer savings of up to 50% until February 15.

     

Rare Disease Day 2026

It's almost time! 
In less than 20 days, the global rare disease community will come together to raise awareness and advocate for equity in health for the 300 million people living with a rare disease. On February 28 2026, groups around the world will organize events for local rare disease communities to come together and raise awareness about their shared challenges.

     
     
   
   

University of Ottawa – Employment Opportunitiy

The University of Ottawa is accepting applications for a Tier 2 Canada Research Chair in neuromuscular disease and related cardiac disorders. This clinical non tenure track position includes appointments with the Department of Medicine, The Ottawa Hospital Research Institute, and the Brain Heart Interconnectome program. 

The deadline to apply is April 4, 2026. 

Stem Cell Network – Training Opportunitiy

Clinical Translation in Cell and Gene Therapy

  • Event Date: March 4, 2026 
  • Time: 1 p.m - 2:30 p.m ET 
  • Location: Virtual (Zoom) 

Call for Experts: IRDiRC Task Force on Digital Twins in Rare Disease Research & Care

The Therapies Scientific Committee (TSC) is launching a Task Force to assess opportunities & barriers for responsible, equitable Digital Twins implementation – landscape/gap analysis ahead!  

Commitment: Monthly 1h calls, 2-day in-person workshop, emails, co-author peer-reviewed papers

Application Deadline: 20th February 2026

Call for Participation: SMA Perspectives Study

Do you or someone you know have spinal muscular atrophy (SMA)? A research team at the University of Alberta is conducting a study on parent, carrier, and patient perspectives on gene therapy, prenatal testing, and family planning in the context of SMA. 

The study involves a short, confidential online survey, and no prior knowledge of gene therapy or prenatal testing is required. Learn more or complete the survey below: 

     
     

Applying for a Grant?  We Can Help!

Share your news & events with the NMD4C

     

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