PARIS, France, March 23, 2023 at 8:30 a.m. (CET) – Pharnext SCA (FR001400BV89 – ALPHA)
(the “Company”), a late-stage biopharmaceutical company developing new therapies for neurodegenerative diseases with no satisfactory therapeutic solution, announces today that data from the CMT&Me digital study on the impact of fatigue in Charcot-Marie-Tooth (CMT) diseases will be presented in the form of a webinar of
45 minutes on 29 March 2023 at 12:00 noon (UK time).
The international digital study CMT&Me aims to better understand the impact of CMT on the daily lives of patients. To date, more than 3,133 patients worldwide are participating in this study.
With the CMT community in the UK contributing extensively to this important patient study, Vitaccess, in partnership with CMT UK and with the support of Pharnext, is hosting a 45 minute webinar to share new results from the CMT&Me app at UK, March 29 at 12:00 p.m. (local time).
Prof. Mario Saporta, Associate Professor of Neurology and Human Genetics at the University of Miami, Miller School of Medicine, and Chairman of the Scientific Council of the CMT&Me study, will comment on these most recent results which particularly concern fatigue and will discuss the impact of CMT on the daily life of patients. This presentation will be followed by a live poll that will open a Q&A session.
To attend this free webinar on the latest results from the CMT&Me study in the UK, you must register before the event by clicking on this LINK.
About the CMT&Me digital study evaluating the lifestyle of patients with CMT
Launched in 2018 over a period of five years in the United States and Europe, CMT&Me is a digital study evaluating the lifestyle of patients with CMTs that allows them to report, on a dedicated application, how this pathology affects their quality of life. life, including their daily pain, mobility and ability to work. The study is conducted by the company Vitaccess in collaboration with patient associations and CMT expert physicians, with the support of Pharnext.
More information on the CMT&Me study: https://clinicaltrials.gov/ct2/show/NCT03782883.
About Charcot-Marie-Tooth disease type 1A (CMT1A)
Charcot-Marie-Tooth (CMT) diseases include a heterogeneous set of peripheral, hereditary, severe, disabling, progressive and chronic neuropathies. CMT1A, the most frequent subtype of CMT, is an orphan disease with a prevalence of 1/5000 affecting around 150,000 people in Europe and the United States, and around 1,500,000 people worldwide. The genetic mutation causing CMT1A is a duplication of the PMP22 gene coding for a peripheral myelin protein. Duplication of this gene induces overexpression of the PMP22 protein and the inability of Schwann cells to produce normal myelin (neural sheath). The myelin sheath thus degraded (structure and functionality) disrupts the conduction of nerve impulses at the level of the peripheral nerves and causes the degradation of the axons. Because of this deterioration of the peripheral nerves, patients suffer from progressive muscular atrophy in the legs and arms leading to problems with walking, running and balance, as well as functional disorders of the hands. They may also suffer from mild to moderate sensory disturbances. The first symptoms usually appear in adolescence and evolve gradually throughout the patient’s life. In the most severe cases, CMT1A patients become dependent on a wheelchair (at least 5% of cases). To date, no curative or symptomatic drug has received marketing authorization for CMT1A. The management of the disease is limited to supportive care such as orthoses, splints, physiotherapy, occupational therapy or surgery.
More information on https://pharnext.com/fr/disease/charcot-marie-tooth
.
About Pharnext
Pharnext is a late-stage biopharmaceutical company developing novel therapies for neurodegenerative diseases that currently have no satisfactory therapeutic solution. Pharnext has a new drug candidate, PXT3003, in development for Charcot-Marie-Tooth disease type 1A (CMT1A), an inherited, rare and debilitating peripheral neuropathy. PXT3003 has orphan drug status in Europe and the United States. In 2018, PXT3003 completed a Phase III clinical study, the PLEO-CMT trial, with encouraging preliminary results. This trial was followed by an open-label extension study, the PLEO-CMT-FU trial, in which 120 patients are still currently continuing treatment with PXT3003. The long-term results suggest a sustained benefit, in terms of tolerance and efficacy, after a total clinical study period of 5 years. A pivotal international Phase III clinical study, the PREMIER trial, is ongoing, in which 387 patients with CMT1A have been enrolled. The first results of the PREMIER trial are expected in the fourth quarter of 2023. PXT3003 was discovered with the R&D approach of Pleotherapy™. Pharnext draws investors’ attention to the risk factors, particularly financial, detailed in its financial reports. More information on
www.pharnext.com
.
Pharnext is listed on the Euronext Growth market in Paris (ISIN code: FR001400BV89).
contacts
|
Financial Press Relations NEWS finance & communication Deborah Schwartz [email protected] +33 (0)1 53 67 36 35 |
Investor Relations NEWS finance & communication Jerome Fabreguettes Leib [email protected] +33 (0)1 53 67 36 78 |
This post has the “? Actusnews SECURITY MASTER” service.
– SECURITY MASTER Key:
l2iblspoZWmVyXFvkpybbpOWamthlZPKaGjKxZKcZcmYm22UlG5pl5XGZnBqlWZn
– To check this key:
https://www.security-master-key.com.
Regulated information:
Inside information:
– Other press releases
Full and original press release in PDF format:
https://www.actusnews.com/news/79011-2023.03.23_webinar-cmtme_en.pdf
© Copyright Actusnews Wire
Receive the company’s next press releases free of charge by email by subscribing to www.actusnews.com