HNF Contributes CMTRN Data to C-Path’s RDCA-DAP

HNF Contributes CMTRN Data to C-Path’s RDCA-DAP, Strengthening Research Capabilities for Inherited Neuropathies

Integration of CMTRN data into RDCA-DAP promises new insights into Charcot-Marie-Tooth disease and related neuropathies, fueling advancements in treatment and care.

TUCSON, Ariz., February 1, 2024 — Critical Path Institute (C-Path) is proud to announce a significant data sharing agreement with the Hereditary Neuropathy Foundation (HNF), integrating valuable data from the Charcot-Marie-Tooth Research Network (CMTRN) into C-Path’s Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®). This integration, facilitated through the National Organization for Rare Disorders’ (NORD) IAMRARE® registry, represents a significant leap forward in the research and understanding for the ultra-rare forms of Charcot-Marie-Tooth (CMT) disease and related inherited neuropathies .

“There are over 130 mutations for the various types of CMT/IN. HNF recognized the gap in supporting the neglected or ultra-rare subtypes of CMT/IN and took a bold step to create a robust natural history study to identify the unique phenotypes associated with each subtype where there were major gaps of information,” said Joy Aldrich, HNF’s Registry Coordinator.

“The collaboration with HNF and the incorporation of the CMTRN data into RDCA-DAP is a transformative step in our mission to tackle rare neurological disorders,” said Alexandre Bétourné, Ph.D., Pharm.D., Executive Director for RDCA-DAP at C-Path. “This partnership broadens the scope of our portfolio and also facilitates an optimized understanding of complex conditions like inherited neuropathies. It’s a leap forward in our journey to accelerate the development of innovative treatment approaches, offering a beacon of hope for individuals and families affected by these conditions.”

Allison Moore, CEO of the Hereditary Neuropathy Foundation, shared her perspective on the collaboration: “At HNF, we are dedicated to making a tangible difference in the lives of those living with CMT and related neuropathies. Contributing our data to RDCA-DAP through NORD’s IAMRARE registry amplifies our impact, allowing us to reach beyond our community and contribute to a global effort in understanding and treating these challenging conditions. Now the CMTRN project has ended, I encourage anyone with any type of CMT to join HNF’s Global Registry for Inherited Neuropathies (GRIN).”

RDCA-DAP, a joint initiative by C-Path and NORD with support from FDA, offers a unique platform promoting cooperation among various stakeholders, including academic institutions, industry leaders, healthcare organizations, and patient advocacy groups. It accelerates the development of treatments for rare diseases through data sharing, analysis, and collaboration.
The CMTRN study included HNPP, CMT2A with optic atrophy, CMT2C, LMNA mediated AD-CMT2, CMT4, CMT4A, 4B1, 1B2 & 4B3, 4C, 4D, 4E, 4F, 4G, 4H, 4J, CMT6, Autosomal Dominant Optic Atrophy ADOA (OPA1) and Giant Axonal Neuropathy (GAN).

The inclusion of CMTRN data into RDCA-DAP advances research specific to CMT/IN and exemplifies the power of collaborative efforts in driving innovative discoveries for the broader rare disease community. It reinforces HNF’s role as a leader in advocating for and supporting CMT/IN research and highlights the potential of shared knowledge in the quest for cures.

Organizations interested in contributing to RDCA-DAP can learn more and reach out at c-path.org/rdca-dap or email [email protected]. The platform is currently open and accepting applications for use; visit portal.rdca.c-path.org for further information.

About Critical Path Institute

Critical Path Institute (C-Path) is an independent, nonprofit established in 2005 as a public-private partnership, in response to the FDA’s Critical Path Initiative. C-Path’s mission is to lead collaborations that advance better treatments for people worldwide. Globally recognized as a pioneer in accelerating drug development, C-Path has established numerous international consortia, programs and initiatives that currently include more than 1,600 scientists and representatives from government and regulatory agencies, academia, patient organizations, disease foundations and pharmaceutical and biotech companies. With dedicated team members located throughout the world, C-Path’s global headquarters is located in Tucson, Arizona and C-Path’s Europe subsidiary is headquartered in Amsterdam, Netherlands. For more information, visit c-path.org.

Critical Path Institute is supported by the Food and Drug Administration (FDA) of the Department of Health and Human Services (HHS) and is 54% funded by the FDA/HHS, totaling $19,436,549, and 46% funded by non-government source(s), totaling $16,373,368. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government.