HNF to Host FDA Patient Listening Session for CMT-SORD
The Hereditary Neuropathy Foundation is hosting an FDA Patient Listening Session for the CMT-SORD community.
FDA Patient Listening Sessions give patients and caregivers a direct line to the agency. The FDA hears firsthand what it means to live with a disease, which symptoms carry the heaviest burden, and what patients need from potential treatments. That input directly informs how the FDA evaluates new therapies.
Why This Session Matters
For rare disease communities, access to the FDA is not guaranteed. Patient Listening Sessions are one of the few formal mechanisms through which lived experience can directly shape regulatory decision-making. When patients speak, the FDA listens, and what is shared in these sessions becomes part of the record that reviewers carry into their evaluation of new treatments. For a condition as under-recognized as CMT-SORD, that visibility is invaluable.
For the CMT-SORD community, this session comes at a critical moment. Earlier this year, Cycle Pharmaceuticals paused all clinical trials and Early Access Programs for govorestat (AT-007) to re-evaluate the data package needed for FDA review. That pause makes the patient voice more important, not less. The FDA needs to understand the full weight of this disease, and this session is a formal opportunity to deliver that message.
What Is CMT-SORD?
CMT-SORD is caused by mutations in the SORD gene that prevent the body from converting sorbitol into fructose. The resulting toxic buildup of sorbitol in peripheral nerve cells leads to progressive muscle weakness, atrophy in the lower legs and hands, and sensory loss. It is one of the most recently identified subtypes of CMT and one of the most promising in terms of treatment potential.
About the Session
The session takes place on Monday, August 4th from 2:00 to 3:30 pm. HNF has selected a handful of CMT-SORD patients, family members, and care partners to share their experiences directly with the FDA. This session is not open to the public.
Opportunities like this one begin with being part of the community. If you are living with CMT and have not yet joined the GRIN Patient Registry, HNF’s global registry for inherited neuropathies, we encourage you to register at joingrin.org so you can be considered for future sessions and advocacy opportunities.
Questions? Contact us at in**@******re.org
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