نتایج جستجو برای: cmt1a

تعداد نتایج: 183  

Journal: :Clinical genetics 2011
G Karadima P Floroskufi G Koutsis D Vassilopoulos M Panas

To the Editor : Charcot–Marie–Tooth type 1 (CMT1), the most frequent hereditary peripheral neuropathy, is characterized by marked clinical and genetic heterogeneity (1). Mutations in genes coding for peripheral myelin protein 22 (PMP22 ; MIM 601097), myelin protein zero (MPZ/P0 ; MIM 159440) and connexin 32 or gap junction beta 1 (Cx32/GJB1 ; MIM 304040) cause CMT1A, CMT1B and CMTX, the most co...

Journal: :Molecules 2017
Mariam Chahbouni María Del Señor López Antonio Molina-Carballo Tomás de Haro Antonio Muñoz-Hoyos Marisol Fernández-Ortiz Ana Guerra-Librero Darío Acuña-Castroviejo

Charcot-Marie-Tooth neuropathy (CMT) is a motor and sensory neuropathy comprising a heterogeneous group of inherited diseases. The CMT1A phenotype is predominant in the 70% of CMT patients, with nerve conduction velocity reduction and hypertrophic demyelination. These patients have elevated oxidative stress and chronic inflammation. Currently, there is no effective cure for CMT; herein, we inve...

2014
Federica Menotti Luca Laudani Antonello Damiani Andrea Macaluso

BACKGROUND Charcot-Marie-Tooth 1A (CMT1A) patients show a reduction of spontaneous activities of daily living measured by means of questionnaires or pedometers, which are quite inaccurate compared to recent measurement techniques. AIM The study aimed at quantifying daily living activities in CMT1A patients by means of inertial sensors, which give information not only on the amount but also on...

2014
Barbara W van Paassen Anneke J van der Kooi Karin Y van Spaendonck-Zwarts Camiel Verhamme Frank Baas Marianne de Visser

PMP22 related neuropathies comprise (1) PMP22 duplications leading to Charcot-Marie-Tooth disease type 1A (CMT1A), (2) PMP22 deletions, leading to Hereditary Neuropathy with liability to Pressure Palsies (HNPP), and (3) PMP22 point mutations, causing both phenotypes. Overall prevalence of CMT is usually reported as 1:2,500, epidemiological studies show that 20-64% of CMT patients carry the PMP2...

Journal: :Journal of medical genetics 1994
B H Holmberg G Holmgren E Nelis C van Broeckhoven B Westerberg

Sixty-seven patients in 29 families with the diagnosis of Charcot-Marie-Tooth disease or hereditary motor and sensory neuropathy in northern Sweden were examined by pedigree and DNA analysis for the CMT1a duplication within chromosome 17p11.2. There were 39 patients in nine families with Charcot-Marie-Tooth type 1 and autosomal dominant inheritance and in all these cases the duplication was see...

2014
Shahram Attarian Jean-Michel Vallat Laurent Magy Benoît Funalot Pierre-Marie Gonnaud Arnaud Lacour Yann Péréon Odile Dubourg Jean Pouget Joëlle Micallef Jérôme Franques Marie-Noëlle Lefebvre Karima Ghorab Mahmoud Al-Moussawi Vincent Tiffreau Marguerite Preudhomme Armelle Magot Laurène Leclair-Visonneau Tanya Stojkovic Laura Bossi Philippe Lehert Walter Gilbert Viviane Bertrand Jonas Mandel Aude Milet Rodolphe Hajj Lamia Boudiaf Catherine Scart-Grès Serguei Nabirotchkin Mickael Guedj Ilya Chumakov Daniel Cohen

BACKGROUND Charcot-Marie-Tooth type 1A disease (CMT1A) is a rare orphan inherited neuropathy caused by an autosomal dominant duplication of a gene encoding for the structural myelin protein PMP22, which induces abnormal Schwann cell differentiation and dysmyelination, eventually leading to axonal suffering then loss and muscle wasting. We favour the idea that diseases can be more efficiently tr...

Journal: :JPMA. The Journal of the Pakistan Medical Association 2015
Shazia Perveen Shazia Mannan Abrar Hussain Sumaira Kanwal

Charcot-Marie-Tooth (CMT) disease is a well-known neural or spinal type of muscular atrophy. It is the most familiar disease within a group of conditions called Hereditary Motor and Sensory Neuropathies (HMSN). The disease was discovered by three scientists several years ago. Several genes are involved as the causative agents for the disease. Hundreds of causative mutations have been found and ...

2017
Rui Wu He Lv Wei Zhang Zhaoxia Wang Yuehuan Zuo Jing Liu Yun Yuan

Charcot-Marie-Tooth 1A (CMT1A) caused by peripheral myelin protein 22 (PMP22) gene duplication is the most common form of hereditary polyneuropathy. Twenty-four genetically confirmed CMT1A patients with sural nerve biopsies were enrolled in this study. The clinical picture included a great variability of phenotype with mean onset age of 22.2 ± 14.5 years (1-55 years). Pathologically, we observe...

2014
Zarife Sahenk Gloria Galloway Kelly Reed Clark Vinod Malik Louise R Rodino-Klapac Brian K. Kaspar Lei Chen Cilwyn Braganza Chrystal Montgomery Jerry R Mendell

Charcot-Marie-Tooth (CMT) neuropathies represent a heterogeneous group of peripheral nerve disorders affecting 1 in 2,500 persons. One variant, CMT1A, is a primary Schwann cell (SC) disorder, and represents the single most common variant. In previous studies, we showed that neurotrophin-3 (NT-3) improved the trembler(J) (Tr(J)) mouse and also showed efficacy in CMT1A patients. Long-term treatme...

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