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December 7, 2018December 7, 2018

FDA Grants Experimental Therapy ACE-083 Fast Track Status for CMT

News

Investigational therapy ACE-083 was granted fast track designation for Charcot-Marie-Tooth disease (CMT) by the United States Food and Drug Administration, Acceleron Pharma recently announced. ACE-083 can prevent muscle shrinkage ... Read more

November 28, 2018November 28, 2018

Modeling of MFN2 Protein Mutations’ Effects May Predict CMT Type 2A’s Severity

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Molecular simulations of the effects of specific mutations in the structure of mitofusin 2 (MFN2) — the key protein altered in Charcot-Marie-Tooth disease type 2A (CMT2A) — may predict the ... Read more

November 16, 2018November 16, 2018

Fontan Surgery for Heart Defects May Not Be Advisable for CMT1A Patients, Case Study Finds

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Patients with Charcot-Marie-Tooth disease (CMT) who undergo Fontan surgery to correct a heart defect in childhood may later develop difficulties with the diaphragm — the muscle that allows us to ... Read more

November 7, 2018November 7, 2018

Certain Gait Scores Can Help Identify CMT and Determine Severity, Study Suggests

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Two scores that evaluate gait characteristics may help differentiate between those who have Charcot-Marie-Tooth (CMT) disease and those who are not affected, according to a study. The scores could ... Read more

October 29, 2018

#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach

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With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Credit ... Read more

October 29, 2018October 29, 2018

CMTA Announces $150,000 Research Challenge Fundraiser for CMT Disease

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To help it reach its fundraising goal by year’s end, the Charcot-Marie-Tooth Association (CMTA) has issued its 35th Anniversary $150,000 Research Challenge. Donations will be matched dollar for dollar through ... Read more

October 23, 2018October 24, 2018

Large Study Focuses on CMT Genetic Variants and Age of Onset in Japan

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Variants in the genes GJB1, MFN2 and MPZ are the predominant causes of Charcot-Marie-Tooth disease (CMT) across Japan, and the first decade of life is the most common onset period ... Read more

October 22, 2018October 22, 2018

Study Details How Balance Issues Affect Children and Adolescents with CMT

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To keep their balance while standing upright, children and adolescents with Charcot-Marie-Tooth (CMT) disease seem to adopt a more stiff posture and sway their bodies less than healthy individuals, researchers ... Read more

October 19, 2018October 19, 2018

International CMT&Me Observational Study to Explore the Impact of CMT Using an App

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A newly launched international observational study is going to explore the overall impact of Charcot-Marie-Tooth disease (CMT) using a tailored app called CMT&Me. Designed by Vitaccess in collaboration with ... Read more

October 17, 2018October 17, 2018

PXT3003 Eases Disability, Found Safe in CMT1A Patients, Phase 3 Trial Shows

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Investigational compound PXT3003 consistently eased disability in patients with mild to moderate Charcot-Marie-Tooth disease type 1A (CMT1A), according to top-line Phase 3 results from Pharnext, which also found that the ... Read more

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