When it comes to developing new treatments, no one understands the impact of Charcot-Marie-Tooth disease type 1A (CMT1A) better than the people living with it. This first-hand knowledge can influence how a therapy is designed, delivered, and made available.
That is the thinking behind a recent survey by CMTA launched through our Patients as Partners in Research program, CMT France, and Nervosave Therapeutics. The organizations partnered to survey 465 people in France and the U.S. to learn more about what it’s like to live with CMT1A and what people want from future treatments.
The results showed some clear differences between participants, notably in symptoms, access to medical care, and treatment priorities.
Symptoms: Same subtype, different experiences
CMT1A is the same genetic condition on both sides of the Atlantic, but the survey revealed differences in how people experience and report their symptoms.
U.S. respondents reported symptoms affecting movement and sensation in both their arms and legs more frequently than their French counterparts. The reasons for this are unclear and may reflect differences in how health care providers identify and track CMT symptoms in the two countries.
Patients say future CMT treatments must be safe, effective and long term
An important finding revealed that regardless of country, people with CMT1A prioritize safety and long-term effectiveness in a new therapy.
Both the French and American respondents wanted therapeutic approaches that directly target the peripheral nerves most affected by CMT1A, while minimizing broader systemic effects on the body. These preferences align with CMTA’s drug delivery research priority, which is focused on getting treatments to cells in the body where they’re most effective.
“It is highly relevant for Nervosave Therapeutics, whose localized gene therapy approach is designed to deliver treatment directly to affected peripheral nerves of arms and legs while minimizing broader systemic exposure,” said Nicolas Tricuad, PhD, Nervosave Co-founder and CEO/CSO. “Nervosave Therapeutics gene therapy also potentially allows for long-term benefit after a single shot treatment.”
Why this kind of research is important
Hearing from 465 people with CMT1A across different countries gives CMTA, CMT France, and Nervosave Therapeutics a clearer picture of what the CMT1A community needs, and a stronger foundation for developing treatments that consider the realities of daily life with CMT.
