Retrophin(美国)

Retrophin

美国Retrophinwww.retrophin.comretrophin，LLC是总部位于纽约的一家私人持有的生物技术公司，侧重于发现和开发为罕见的危及生命的疾病的治疗。retrophin目前正在开发治疗杜氏肌营养不良症，脊髓性肌萎缩症，囊肿性纤维化和myotubular肌病。retrophin的铅内部发现的化合物，RE-001，是杜氏肌营养不良蛋白替代疗法。retrophin打算初步发展为罕见 的肾病，局灶节段性肾小球硬化的药物。Retrophin,LLCisaprivately-heldNewYork-based,biotechnologycompanyfocusedondiscoveringanddevelopingtreatmentsforrareandlife-threateningdiseases.RetrophiniscurrentlydevelopingtreatmentsforDuchennemusculardystrophy,spinalmuscularatrophy,cysticfibrosisandmyotubularmyopathy.Retrophin'sleadinternallydiscoveredcompound,RE-001,isaproteinreplacementtherapyforDuchennemusculardystrophy.Retrophinintendstoinitiallydevelopthedrugforrarenephropathies,includingFocalSegmentalGlomerulosclerosis.