Concerned about the rare disease "Red Blood Cell-producing Prosofe and X-Chain Dystrophy": Launched Global Phase III Clinical Trials of MT-7117
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Last Update: 2020-06-25
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Source: Internet
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Author: User
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Mitsubishi Tianbian Pharmaceutical Co., Ltdannounced today that it has begun a global Phase III clinical trial of MT-7117, a selective black skin quality 1 receptor (MC1R) agonisant, aimed at treating patients with phototoxic history of red cell-generated protogenide and X-chain dysenteryred blood cell-generated primary rickets (EPP) is a form of ricketsRickets is caused by abnormal lycopene productionEPP is caused by a disease-causing mutation in the FECH gene, which causes the activity of hemoglobin-producing enzyme (FECH) (an important enzyme in the process of hemoglobin production), which in turn causes the accumulation of protoscosis in the bone marrow, red blood cells, plasma, skin and liverThe accumulation of the original radon can cause the patient to be extremely sensitive to daylightMT-7117 was granted fast-track by the U.SFDAin 2018 and was certified as an orphan drug on June 8, 2020The Phase II Proof of Concept Study (ENDEAVOR) of MT-7117 showed that patients in the MT-7117 group significantly improved their sun exposure-related symptoms between 1 hour after sunrise and 1 hour before sunset, and safety was within acceptable limits, compared to the 100mg placebo group
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