New Task Force to Address Challenges in LGMD Research

A new task force entrusted with advancing therapeutic development for limb-girdle muscular dystrophies (LGMDs) has been formed by the Critical Path Institute (C-Path) under its Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP^®).

Leading organizations and specialists come together as part of this initiative to address the difficulties involved in the development of drugs for LGMDs. A class of diseases known as LGMDs affects the voluntary muscles, mainly the hip and shoulder regions.

The LGMD2D Foundation, the CureLGMD2i Foundation, the Jain Foundation, ML Bio Solutions, the Coalition to Cure Calpain 3, the LGMD2i Research Fund, and The Speak Foundation are among the task force's confirmed members.

Furthermore, partnerships with influential think tanks and patient advocacy organizations are underway to guarantee thorough stakeholder involvement.

This task force will use the members' combined expertise to hasten the creation of novel treatments for LGMDs. Through C-Path’s RDCA-DAP, an FDA-funded initiative that offers a centralized and standardized infrastructure to support and accelerate rare disease characterization, the task force will integrate patient-level data from various sources to identify and address critical gaps in the research and development of LGMDs.

The partnership increases the effectiveness of therapeutic breakthroughs for LGMDs by offering a neutral forum for relevant discussion and data sharing amongst stakeholders from industry, academia, regulatory bodies, and the LGMDs community.

Limb-girdle muscular dystrophies are a devastating group of diseases with significant unmet medical needs. By convening this task force, we are harnessing the collective strengths and knowledge of our members to drive forward the development of effective treatments for those affected by LGMDs.”

Alexandre Betourne, RDCA-DAP Executive Director, Critical Path Institute

Doug Sproule, M.D., M.Sc., Chief Medical Officer at ML Bio Solutions, commented on the task force’s formation: “We are grateful for the opportunity to join the LGMDs Task Force to enhance collaboration between key stakeholders and advance the development of potential treatments for LGMDs, where there currently is a large unmet need. By providing our LGMD2I/R9 natural history data, we hope to increase the understanding of the progressive condition to improve the lives of those with LGMDs and their families.”

The founder of the Speak Foundation, Kathryn Bryant Knudson, emphasized the transformative potential of collaboration: “At the Speak Foundation, we understand that collaboration and shared knowledge are the cornerstones of groundbreaking drug development. This task force will harness data to forge powerful tools essential for innovation.”

Heidi Grabenstatter, RDCA-DAP Scientific Director, Critical Path Institute concluded, “I look forward to leading this effort with my colleagues from RDCA-DAP and Dr. Ramona Belfiore-Oshan, Executive Director for the Duchenne Regulatory Science Consortium (D-RSC). Leveraging data from multiple sources and across LGMD subtypes in addition to collaborative knowledge sharing within the task force will be key to developing a clinically impactful drug developmental tool that helps accelerate therapies for patients living with high unmet needs in the LGMD community.”