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

Orphanet Journal of Rare Diseases

Volumn 9, Issue 1, 2014, Pages

  Chumakov, Ilya ^a   Milet, Aude ^a   Cholet, Nathalie ^a   Primas, Gwenaël ^a   Boucard, Aurélie ^a   Pereira, Yannick ^a   Graudens, Esther ^a   Mandel, Jonas ^a   Laffaire, Julien ^a   Foucquier, Julie ^a   Glibert, Fabrice ^a   Bertrand, Viviane ^a   Nave, Klaus Armin ^b   Sereda, Michael W ^b,c   Vial, Emmanuel ^a   Guedj, Mickaël ^a   Hajj, Rodolphe ^a   Nabirotchkin, Serguei ^a   Cohen, Daniel ^a  

^a Pharnext 11 ^*  (France)

^b MAX PLANCK INSTITUTE OF EXPERIMENTAL MEDICINE   (Germany)

^c UNIVERSITY MEDICAL CENTER GÖTTINGEN   (Germany)

Author keywords

Baclofen; CMT1A; Combination therapy; Low dose; Naltrexone; Repurposing; Sorbitol; Synergy; Systems Biology

Indexed keywords

BACLOFEN; MYELIN BASIC PROTEIN; NALTREXONE; PERIPHERAL MYELIN PROTEIN 22; PXT 3003; SORBITOL; UNCLASSIFIED DRUG; MYELIN PROTEIN; PMP22 PROTEIN, RAT;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CHARCOT MARIE TOOTH DISEASE TYPE 1A NEUROPATHY; CONTROLLED STUDY; DORSAL ROOT; DRUG EFFICACY; DRUG POTENCY; DRUG SAFETY; GENE OVEREXPRESSION; HEREDITARY MOTOR SENSORY NEUROPATHY; IN VITRO STUDY; IN VIVO STUDY; MALE; MOTOR NERVE CONDUCTION; MOUSE; MUSCLE STRENGTH; MYELINATION; NERVE CONDUCTION; NERVE CRUSH; NEUROPATHY; NEUROPROTECTION; NONHUMAN; PLEIOTROPY; PROTEIN FOLDING; RAT; REMYELINIZATION; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SCHWANN CELL; SCIATIC NERVE; TREATMENT DURATION; WEIGHT BEARING; ANIMAL; ANTAGONISTS AND INHIBITORS; AXON; BIOSYNTHESIS; CHARCOT-MARIE-TOOTH DISEASE; COCULTURE; COMBINATION DRUG THERAPY; DISEASE MODEL; DOWN REGULATION; DRUG EFFECTS; DRUG REPOSITIONING; FEMALE; GENE EXPRESSION REGULATION; METABOLISM; MYELINATED NERVE; PATHOLOGY; PHYSIOLOGY; PROCEDURES; SCIATIC NEUROPATHY; SPRAGUE DAWLEY RAT; TRANSGENIC RAT;

ANIMALS; AXONS; BACLOFEN; CHARCOT-MARIE-TOOTH DISEASE; COCULTURE TECHNIQUES; DISEASE MODELS, ANIMAL; DOWN-REGULATION; DRUG REPOSITIONING; DRUG THERAPY, COMBINATION; FEMALE; GENE EXPRESSION REGULATION; MALE; MICE; MYELIN PROTEINS; NALTREXONE; NERVE FIBERS, MYELINATED; RATS; RATS, SPRAGUE-DAWLEY; RATS, TRANSGENIC; SCIATIC NEUROPATHY; SORBITOL;

EID: 84924985851     PISSN: None     EISSN: 17501172     Source Type: Journal    
DOI: 10.1186/s13023-014-0201-x     Document Type: Article

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