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PXT3003

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https://www.readbyqxmd.com/read/27387831/erratum-to-an-exploratory-randomised-double-blind-and-placebo-controlled-phase-2-study-of-a-combination-of-baclofen-naltrexone-and-sorbitol-pxt3003-in-patients-with-charcot-marie-tooth-disease-type-1a
#1
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
No abstract text is available yet for this article.
2016: Orphanet Journal of Rare Diseases
https://www.readbyqxmd.com/read/26764298/-therapy-for-charcot-marie-tooth-disease-from-the-standpoint-of-neurologists
#2
REVIEW
Masanori Nakagawa
To date, there is no approved pharmacologic treatment for any form of Charcot-Marie-Tooth disease (CMT). However, some clinical or preclinical trials for CMT1A have been undertaken, for example Neurotrophin-3, PXT3003, and neuregulin-1. Gene therapy for CMT1X, CMT2F and Giant axonal neuropathy using animal model or culture cells have been reported with some interesting results. Stem cell research for example iPS cells derived from patients with CMT2A or CMT2E, is being conducted to clarify the mechanism of CMT and find therapeutic clues...
January 2016: Brain and Nerve, Shinkei Kenkyū No Shinpo
https://www.readbyqxmd.com/read/26070802/a-meta-analysis-of-randomized-double-blind-clinical-trials-in-cmt1a-to-assess-the-change-from-baseline-in-cmtns-and-onls-scales-after-one-year-of-treatment
#3
LETTER
Jonas Mandel, Viviane Bertrand, Philippe Lehert, Shahram Attarian, Laurent Magy, Joëlle Micallef, Ilya Chumakov, Catherine Scart-Grès, Mickael Guedj, Daniel Cohen
CMT1A is the most common inherited peripheral neuropathy. There is currently no approved treatment. We performed a meta-analysis including four randomized, double-blind, Placebo-controlled clinical trials to assess the disease progression after one year under Placebo, Ascorbic Acid (AA) or PXT3003, a combination of three repurposed drugs. We observed a weak deterioration in patients under Placebo, well below the reported natural disease progression. Patients treated with AA were stable after one year but not significantly different from Placebo...
2015: Orphanet Journal of Rare Diseases
https://www.readbyqxmd.com/read/25519680/an-exploratory-randomised-double-blind-and-placebo-controlled-phase-2-study-of-a-combination-of-baclofen-naltrexone-and-sorbitol-pxt3003-in-patients-with-charcot-marie-tooth-disease-type-1a
#4
RANDOMIZED CONTROLLED TRIAL
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 treated when targeting multiple disease-relevant pathways. In CMT1A patients, we therefore tested the potential of PXT3003, a low-dose combination of three already approved compounds (baclofen, naltrexone and sorbitol)...
December 18, 2014: Orphanet Journal of Rare Diseases
https://www.readbyqxmd.com/read/25491744/polytherapy-with-a-combination-of-three-repurposed-drugs-pxt3003-down-regulates-pmp22-over-expression-and-improves-myelination-axonal-and-functional-parameters-in-models-of-cmt1a-neuropathy
#5
Ilya Chumakov, Aude Milet, Nathalie Cholet, Gwenaël Primas, Aurélie Boucard, Yannick Pereira, Esther Graudens, Jonas Mandel, Julien Laffaire, Julie Foucquier, Fabrice Glibert, Viviane Bertrand, Klaus-Armin Nave, Michael W Sereda, Emmanuel Vial, Mickaël Guedj, Rodolphe Hajj, Serguei Nabirotchkin, Daniel Cohen
Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common inherited sensory and motor peripheral neuropathy. It is caused by PMP22 overexpression which leads to defects of peripheral myelination, loss of long axons, and progressive impairment then disability. There is no treatment available despite observations that monotherapeutic interventions slow progression in rodent models. We thus hypothesized that a polytherapeutic approach using several drugs, previously approved for other diseases, could be beneficial by simultaneously targeting PMP22 and pathways important for myelination and axonal integrity...
2014: Orphanet Journal of Rare Diseases
https://www.readbyqxmd.com/read/25454638/hereditary-motor-and-sensory-neuropathies-or-charcot-marie-tooth-diseases-an-update
#6
REVIEW
Meriem Tazir, Tarik Hamadouche, Sonia Nouioua, Stephane Mathis, Jean-Michel Vallat
Hereditary motor and sensory neuropathies (HMSN) or Charcot-Marie-Tooth (CMT) diseases are the most common degenerative disorders of the peripheral nervous system. However, the frequency of the different subtypes varies within distinct populations. Although more than seventy clinical and genetic forms are known to date, more than 80% of CMT patients in Western countries have genetic abnormalities associated with PMP22, MPZ, MFN2 and GJB1. Given the considerable genetic heterogeneity of CMT, we emphasize the interest of both clinical and pathological specific features such that focused genetic testing could be performed...
December 15, 2014: Journal of the Neurological Sciences
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