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Preparations for clinical trials of unfolded protein response (UPR) inhibitors (such as Sephin1) that target the upregulated UPR in patients with Charcot–Marie–Tooth disease (CMT) carrying MPZ mutations are currently underway.… Click to show full abstract
Preparations for clinical trials of unfolded protein response (UPR) inhibitors (such as Sephin1) that target the upregulated UPR in patients with Charcot–Marie–Tooth disease (CMT) carrying MPZ mutations are currently underway. The inclusion criteria for these trials are still being formulated. Our objective was to characterize the relation between genotypes and phenotypes in patients with CMT caused by MPZ mutations, and to refine the inclusion criteria for future trials.
Keywords:
genotype phenotype;
phenotype correlation;
myelin protein;
french patients;
correlation french;
patients myelin
Journal Title: European Journal of Neurology
Year Published: 2021
Link to full text (if available)
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Journal Title: European Journal of Neurology
Year Published: 2021
Link to full text (if available)
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recommendations!