Pharnext: publication of data on the impact of symptoms of Charcot-Marie-Tooth disease type 1A on the daily lives of patients

» Pharnext: publication of data on the impact of symptoms of Charcot-Marie-Tooth disease type 1A on the daily lives of patients MyPharma Editions

Posted on Thursday September 15th, 2022

Pharnext, a late-stage biopharmaceutical company developing new therapies for neurodegenerative diseases with no satisfactory therapeutic solution, announced the publication of data on the impact of symptoms of Charcot-Marie-Tooth disease type 1A (CMT1A) on the daily life of patients, from the CMT&Me digital study, in the Journal of Clinical Neuromuscular Disease (JCNMD).

CMT&Me is an observational, non-interventional digital study, launched in October 2018 in Europe and the United States to assess the lifestyle of patients with CMT over a period of 5 years. The study regularly collects information provided directly by patients, all CMT subtypes combined, including CMT1A. Its objective is to better understand the impact of the disease on their daily lives and to help them manage their disease and their treatments. In addition, it also aims to educate various audiences about CMTs and help in the evaluation of potential new treatments. This study is being conducted by the company Vitaccess in collaboration with patient associations and expert physicians from CMTs, with the support of Pharnext.

The data show that in patients with CMT subtype 1A (CMT1A patient), the reported impact of symptoms is high with difficulty using their limbs, fatigue, pain, and impaired quality of life. Perceived severity seems to vary in the cohort of CMT1A patients considered, which can be explained by differences in prescribed, drug or functional rehabilitation treatments, but also by disparities in the different health systems and practices of a country. to the other.

The most important symptoms reported are weakness in the extremities (hands and feet), difficulty walking and fatigue. Nearly half of study participants saw the severity of their symptoms worsen since their diagnosis. Anxiety and depression were reported by more than a third of study participants, and the use of functional rehabilitation therapies, drug treatments, wearing splints or other walking aids was high.

Prof Florian P. Thomas, MD, MA, PhD, MS, Founding Chairman and Professor, Department of Neuroomige at Hackensack Meridian School of Medicine, Hackensack (NJ, USA) and Principal Investigator of the PLEOCMT trial in the US states: “ These first data from the digital study, CMT&Me, on the lifestyle of patients help to better understand the consequences of this debilitating and progressive disease, for which there is currently no specific approved treatment. These data demonstrate the high and unmet medical need caused by the symptoms of CMT1A, and support previous studies on the natural history of the disease. They provide more precise information to those who follow the progression of their disease, to enable them to exchange easily with their neurologists and caregivers. They also underline the need to educate patients and their carers: for patients, to give them the means to express their needs in terms of care, and for health professionals to become aware of the need for care. multidisciplinary taking into account the entire disease including occupational therapists, foot and ankle surgeons and psychologists. »

The article titled “Patient-Reported Symptom Burden of Charcot–Marie–Tooth Disease Type 1A: Findings From an Observational Digital Lifestyle Study” is available online here.

Source and visual: Pharnext


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