Charcot-Marie-Tooth Association Company Profile

15:51 EDT 25th June 2018 | BioPortfolio

News Articles [606 Associated News Articles listed on BioPortfolio]

Families support groundbreaking research on Charcot-Marie-Tooth disease

Lorena Martin's twin daughters kick butt on the soccer field. Martin is proud of how they hold their own, despite having a rare genetic disease called Charcot-Marie-Tooth (CMT), which has damaged thei...

Defect in debilitating Charcot-Marie-Tooth reversed in mouse nerves

Scientists have developed a new drug compound that sho

Flex Pharma to cut two trials, reduce headcount and explore merger options

Oral tolerability concerns have prompted the US biotech to close Ph II clinical trial investigations of FLX-787 in amyotrophic lateral sclerosis (ALS) and Charcot-Marie-Tooth (CMT).

Scientists zero in on treatment for Charcot-Marie-Tooth disease

Scientists have now shown a path to developing treatments for disease subtype CMT2D.

Pharnext präsentiert neueste Forschungsdaten zu zwei seiner führenden Produktkandidaten bei mehreren internationalen wissenschaftlichen Tagungen

Phase-III-Studie-Ergebnisse zum Arzneimittelkandidat PXT3003 zur Behandlung von Charcot-Marie-Tooth Typ 1A werden in sechs Posterpräsentationen und zwei mündlichen Präsentationen vorgestellt Arzne....

Pharnext will Topline-Ergebnisse der zulassungsrelevanten Phase-3-Studie zu PXT3003 zur Behandlung der Charcot-Marie-Tooth-Krankheit Typ 1A bis Oktober 2018 vorlegen

Pharnext SA (Paris:ALPHA) (FR0011191287 ALPHA), ein biopharmazeutisches Unternehmen und Vorreiter eines neuen Ansatzes zur Entwicklung innovativer Medikamentenkombinationen, die auf Big Data aus d...

TSRI researchers show path to developing treatments for Charcot-Marie-Tooth disease

Scientists at The Scripps Research Institute have now shown a path to developing treatments for disease subtype CMT2D.

Pharnext's Charcot-Marie-Tooth 1A disease drug nabs priority review in China

China's State Drug Administration has given priority review status to Pharnext's new drug application for PXT3003, which is b -More- 

PubMed Articles [1660 Associated PubMed Articles listed on BioPortfolio]

Whole-Genome Linkage Analysis with Whole-Exome Sequencing Identifies a Novel Frameshift Variant in NEFH in a Chinese Family with Charcot-Marie-Tooth 2: A Novel Variant in NEFH for Charcot-Marie-Tooth 2.

Charcot-Marie-Tooth disease (CMT) is the most common neurodegenerative disorder of the peripheral nervous system. More than 50 genes/loci were found associated with the disease. We found a family with...

Electromyographic and biomechanical analysis of step negotiation in Charcot Marie Tooth subjects whose level walk is not impaired.

Charcot-Marie-Tooth (CMT) is a slowly progressive disease characterized by muscular weakness and wasting with a length-dependent pattern. Mildly affected CMT subjects showed slight alteration of walki...

A role for the GDAP1 gene in the molecular pathogenesis of Charcot‑Marie‑Tooth disease.

In 2002 a series of mutations in the GDAP1 gene were reported in patients suffering from Charcot‑Marie‑Tooth disease manifesting as early-onset, progressive distal‑muscle wasting and weakness. T...

Gait and footwear in children and adolescents with Charcot-Marie-Tooth disease: A cross-sectional, case-controlled study.

Children with Charcot-Marie-Tooth disease (CMT) report problems with gait and footwear. We evaluated differences in spatio-temporal gait variables and gait variability between children with CMT and ty...

The spectrum of Charcot-Marie-Tooth disease due to myelin protein zero: An electrodiagnostic, nerve ultrasound and histological study.

Nerve ultrasound (US) data on myelin protein zero (MPZ)-related Charcot-Marie-Tooth disease (CMT) are lacking. To offer a comprehensive perspective on MPZ-related CMTs, we combined nerve US with clini...

Clinical Trials [1943 Associated Clinical Trials listed on BioPortfolio]

Charcot-Marie-Tooth Disease (CMT) Infant Scale (INC-6611)

The purpose of this study is to develop and validate a clinical outcome measure to evaluate disability and disease progression of children 3 years of age and younger (infants and toddlers)...

Effects of Coenzyme Q10 on Charcot-Marie-Tooth Disease

The object of this research is to test the effectiveness of Coenzyme Q10 (CoQ10) on symptoms of weakness, fatigue, and pain in persons with Charcot-Marie-Tooth disease (CMT).In this study ...

Follow up and Observation of Charcot Marie Tooth Disease in Families

The study is aimed to test the hypothesis that there is anticipation in CMT

Study of ACE-083 in Patients With Charcot-Marie-Tooth Disease

This is a multicenter, phase 2 study to evaluate the safety, tolerability, pharmacodynamics (PD), efficacy, and pharmacokinetics (PK) of ACE-083 in patients with CMT1 and CMTX, to be condu...

Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A

All randomised patients with Charcot-Marie-Tooth Type 1A (CMT1A) who completed the primary study CLN-PXT3003-02, i.e. 15-month double-blind treatment with PXT3003 or placebo, will be eligi...

Companies [1308 Associated Companies listed on BioPortfolio]

Charcot-Marie-Tooth Association

Hereditary Neuropathy Foundation

Hereditary Neuropathy Foundation (HNF) is a non-profit 501(c)3 organization whose mission is to increase awareness and accurate diagnosis of Charcot-Marie-Tooth disease (CMT) and ...

Pharnext SAS

Pharnext is an advanced clinical stage biopharmaceutical company founded by renowned scientists and entrepreneurs including Professor Daniel Cohen, a pioneer in modern genomics. P...

Ella Marie Foundation

The Ella Marie Foundation was founded in 1998 with a simple desire of helping others. Over the past 16 years, that mission has expanded into a cohesive effort to enhance the lives...

Marie Claire

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