Retrophin(美國)

Retrophin

美國Retrophinwww.retrophin.comretrophin，LLC是總部位于紐約的一家私人持有的生物技術公司，側重于發(fā)現和開發(fā)為罕見的危及生命的疾病的治療。retrophin目前正在開發(fā)治療杜氏肌營養(yǎng)不良癥，脊髓性肌萎縮癥，囊腫性纖維化和myotubular肌病。retrophin的鉛內部發(fā)現的化合物，RE-001，是杜氏肌營養(yǎng)不良蛋白替代療法。retrophin打算初步發(fā)展為罕見 的腎病，局灶節(jié)段性腎小球硬化的藥物。Retrophin,LLCisaprivately-heldNewYork-based,biotechnologycompanyfocusedondiscoveringanddevelopingtreatmentsforrareandlife-threateningdiseases.RetrophiniscurrentlydevelopingtreatmentsforDuchennemusculardystrophy,spinalmuscularatrophy,cysticfibrosisandmyotubularmyopathy.Retrophin'sleadinternallydiscoveredcompound,RE-001,isaproteinreplacementtherapyforDuchennemusculardystrophy.Retrophinintendstoinitiallydevelopthedrugforrarenephropathies,includingFocalSegmentalGlomerulosclerosis.