We are thrilled to announce a new clinical workshop focused on establishing subtype-specific care recommendations for LAMA2-RD, and using this experience as a prototype to develop recommendations for four other subtypes of congenital muscular dystrophy (Collagen 6-RD, Dystroglycanopathy, LMNA-CMD, SELENON-RM).
On the initiative of Voor Sara and Cure CMD, and thanks to the great work of our organizers, the European Neuromuscular Centre (ENMC) has approved our application to host an international workshop in January 2025, in The Netherlands.
We are very proud and grateful to our expert neuromuscular organizers for leading the ENMC application submission:
Dr. Reghan Foley, United States
Dr. Andrea Klein, Switzerland
Dr. Anna Sarkozy, United Kingdom
Dr. Alberto Zambon, Italy
Dr. Nicol Voermans, The Netherlands
Workshop organizers, Voor Sara, and Cure CMD met throughout early 2024 to determine the aims and scope of the workshop, prioritizing the unmet needs of the affected community related to informed and proactive care. Discussions have centered around how best to determine the issues most relevant to affected individuals and their caregivers, ensuring that clinicians with CMD expertise from many countries are involved in this process. Deliverables include publication of care recommendations that are widely available to the international community. We have set the following aims for this project:
Gather international multidisciplinary neuromuscular specialists to summarize evidence for care recommendations in LAMA2-RD, and identify where evidence is currently lacking;
Define the most appropriate methodology to reach consensus for clinical care recommendations based on collective international expertise for LAMA2-RD by inclusion of the wider global community, given that LAMA2-RD is a rare condition with limited clinical care evidence;
Once complete, use the process as a prototype for the development of similar publications for Collagen 6-RD, Dystroglycanopathy, LMNA-CMD, and SELENON-RM.
We have composed an invitation list of outstanding experts from around the world, and have developed a challenging program focused on making the most out of this process and delivering the best care guidance possible. We are grateful to ENMC for this opportunity, and look forward with anticipation to a successful workshop.
Stay tuned for information in the coming weeks about how the LAMA2 affected community can contribute to the success of this project by sharing their perspectives!