Pharnext SA announced the publication of symptom burden findings of Charcot-Marie-Tooth Disease Type1A ("CMT1A") from the ‘Real-World' Digital Lifestyle Study, CMT&Me, in the Journal of Clinical Neuromuscular Disease ("JCNMD"). CMT&Me is a real-world, observational, non-interventional, digital lifestyle study launched in October 2018 for a duration of 5 years, both in Europe and the US, where self-reported data from patients with CMT, all subtypes including CMT1A, are collected on a regular basis. The objective of the study is to better understand the impact of the disease on patients' daily lives and help them manage their condition and treatment, as well as raise awareness and assess the value of potential new treatments.

This study is managed by the company Vitaccess in collaboration with patient advocacy groups and key opinion leaders in the field, with the support of Pharnext. The findings on patients with the 1A subtype of CMT (CMT1A) showed that patient-reported symptoms burden is high, with study participants' registering difficulties using limbs, fatigue, pain, and impaired quality of life. Burden severity appears to differ across the population, possibly driven by differences in rehabilitative and prescription-based interventions, and country-specific health care variability.

Symptoms ranked with highest importance were weakness in the extremities, difficulty in walking, and fatigue. Almost half of study participants experienced a worsening of symptom severity following diagnosis. Anxiety and depression were each reported by over one-third of participants in the study and use of rehabilitative interventions, medications, and orthotics or walking aids was high.