MICYRN Secures $20 Million Grant from CIHR for a Pediatric Rare Disease Clinical Trials and Treatment Network
The NMD4C are excited to share MICYRN‘s success in securing a $20 million grant from CIHR Institute of Genetics to support the development and execution of RareKids-CAN: Pediatric Rare Disease Clinical Trials and Treatment Network. Under the leadership of MICYRN’s Scientific Director, and Nominated Principal Applicant, Dr. Thierry Lacaze-Masmonteil, RareKids-CAN is a transformative initiative set to revolutionize the landscape of rare disease research and treatment for children and their families in Canada and beyond.
At the core of RareKids-CAN is a coalition of diverse national and international experts including patient/family partners and patient organizations pooling their collective knowledge and experience in the pediatric rare disease community. Leveraging the strengths of MICYRN and its 17 maternal/child health research institutions, aimed at streamlining complex, pediatric, multi-center, multi-jurisdictional clinical trials, RareKids-CAN is primed for swift and effective operationalization.
RareKids-CAN aims to establish a robust platform in Canada to support pediatric rare disease clinical trials, providing essential infrastructure and resources. It seeks to enhance capacity through comprehensive training and mentorship opportunities, attract international trials and investments, foster inclusivity, and improve accessibility. The network prioritizes data collection and analysis to inform decision-making, while also supporting increased submissions for Health Canada authorization, ensuring timely access to innovative therapies for those impacted by rare diseases.
NMD4C Investigators involved with RareKids-CAN network grant
The NMD4C is excited to support this initative – we were pleased to share a letter of support for the grant application, and celebrate the many NMD4C investigators who are a part of the RareKids-CAN network:
- Dr. Leanne Ward (Executive Committee Member)
- Dr. Alexandra King (Executive Committee Member)
- Dr. Craig Campbell (Executive Committee Member)
- Dr. Maryam Oskoui (Principal Applicant, Co-Lead of Health Economics, HTA and Reimbursement Sub-Platform)
- Dr. Malcolm King (Co-Applicant, Working group member)
- Dr. Homira Osman (Co-Applicant, Working group member)
- Dr. Hanns Lochmüller (Co-Applicant)
- Dr. James Dowling (Co-Applicant)
- Stacey Lintern (Collaborator, Working group member)
For more information, please visit MICYRN’s website.
Read next...
Fourth Annual Clinical & Postdoctoral Fellowship Competition from NMD4C, MDC to Open Fall 2024
NMD4C together with Muscular Dystrophy Canada are excited to announce that our annual Neuromuscular Fellowship Competition will open again for a fourth year on September 3, 2024!
NMD4C Wraps up Second Year of Training Series for Canadian Neuromuscular Clinical Fellows
We’re excited to share that the second annual National Neuromuscular Lecture Series (NNLS) finished last week!
NMD4C Host Inaugural Basic Research Summer School
Over May 27-28 2024, the NMD4C animal and cell-based research team hosted the first-ever annual basic research summer school for neuromuscular research trainees at McGill University in Montréal, QC.
NMD4C Investigator Dr. Reshma Amin Leads Successful CIHR Grant Application to Establish a Framework for Real-World Evidence for Canadian Neuromuscular Diseases
Real-World Evidence for Canadian Neuromuscular Disease: Establishing a Framework for National Integration of Patient Reported Outcomes, Clinical Registry Data, Healthcare Utilization and Healthcare Associated Costs The NMD4C is excited to share that network investigator Dr. Reshma Amin has been awarded funding from the CIHR to establish a framework for real-world evidence (RWE) for Canadian…
Spinal Muscular Atrophy Outcome Measures Workshop (English Language)
The NMD4C will be hosting an English-language interactive training workshop on standardizing assessment practices in SMA outcome measures.
NMD4C Launch Survey to Assess Familiarity and Experience in Outcome Measures in NMDs
As we anticipate novel treatments for other neuromuscular conditions, we would like to understand your familiarity and experience with the outcome measures in clinical trials underway or for treatments for NMDs approved in other jurisdictions through a short survey.