Pharnext kondigt resultaat van onderzoek naar Charcot-Marie-Tooth Neuropathy Score aan
PARIJS–(BUSINESS WIRE)– Pharnext SA (Paris:ALPHA) (FR00111911287 – ALPHA), een Frans biofarmaceutisch bedrijf met een geavanceerd portfolio op gebied van neurodegeneratieve ziekten, heeft vandaag bekendgemaakt dat PLoS One de statistische resultaten heeft gepubliceerd van een onderzoek naar de toepassing van de klinische schaalindeling Charcot-Marie-Tooth Neuropathy Score (CMTNS) bij de ziekte Charcot-Marie-Tooth 1A (CMT1A).
CMTNS is de eerste schaalindeling die belemmeringen en het ziekteverloop bij CMT-patiënten kwantificeert en meet. Het systeem is bij het merendeel van alle voltooide klinisch-wetenschappelijke onderzoeken naar CMT1A de toegepast als primair eindpunt. Het vermogen een respons op behandelingen te meten is echter nog niet bewezen.
In het artikel “A Rasch Analysis of the Charcot-Marie-Tooth Neuropathy Score (CMTNS) in a Cohort of Charcot-Marie-Tooth Type 1A Patients” schrijven Wenjia Wang en andere onderzoekers dat CMTNS bijzonder nuttig is voor het meten van de ernst van CMT1a. CMTNS is daarentegen minder precies bij de beoordeling van milde varianten van de ziekte. Daarom raden de auteurs aan de schaalindeling verder te verfijnen, zodat ze de therapeutische effectiviteit bij alle vormen van CMT1A kunnen meten: van mild tot matig en ernstig.
Pharnext Announces Findings Evaluating Charcot-Marie-Tooth Neuropathy Score |
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PARIS–(BUSINESS WIRE)– Pharnext SA (Paris:ALPHA) (FR00111911287 – ALPHA), a French biopharmaceutical company developing an advanced portfolio of products in the field of neurodegenerative diseases, announced today publication in PLoS One of statistical findings evaluating the clinical scale Charcot-Marie-Tooth Neuropathy Score (CMTNS) for application in Charcot-Marie-Tooth Disease Type 1A (CMT1A). CMTNS is the first clinical scale dedicated to quantify impairment and measure disease progression in CMT patients. It has been used as the primary endpoint in most completed clinical trials for CMT1A to date. However, its ability to measure responses to treatment has not yet been demonstrated. In the article titled: “A Rasch Analysis of the Charcot-Marie-Tooth Neuropathy Score (CMTNS) in a Cohort of Charcot-Marie-Tooth Type 1A Patients” authors Wenjia Wang et al. report that CMTNS remains particularly useful for measuring disease severity in CMT1A. However, CMTNS appears less sensitive in assessing mild forms of the disease. Therefore, the authors recommend further refinement of the scale to better assess therapeutic efficacy in all forms of CMT1A: mild, moderate and severe. Daniel Cohen, M.D., Ph.D., Co-Founder and Chief Executive Officer of Pharnext, said “ One of the major challenges of CMT1A trial design is selecting a clinically meaningful endpoint. The findings confirm the limitations of the CMTNS. They support Pharnext’s decision, endorsed by the European and American regulatory agencies, not to use CMTNS as the primary efficacy endpoint in our pivotal PLEO-CMT trial for patients suffering from mild to moderate forms of CMT1A, and to use the Overall Neuropathy Limitations Scale (ONLS) instead. The ONLS has been used as the primary endpoint to assess treatment efficacy in other peripheral neuropathies pivotal trials.” The publication can be found here: http://journals.plos.org/plosone/article?id=10.1371/journal.pone.0169878 About CMT1A To date, no curative or symptomatic medications have been approved and treatment consists of supportive care such as orthotics, leg braces, physical and occupational therapy or surgery. About PLEO-CMT Trial For more information about the PLEO-CMT clinical trial, please visit the following website: About Pharnext The company Pharnext is listed on Euronext Alternext Stock Exchange in Paris (ISIN code: FR00111911287). For more information, visit www.pharnext.com PLEODRUG © and PLEOTHERAPY © are registered trademarks by Pharnext View source version on businesswire.com: http://www.businesswire.com/news/home/20170201005935/en/ Contacts Pharnext |