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美國Retrophin
retrophin，LLC是總部位于紐約的一家私人持有的生物技術(shù)公司，側(cè)重于發(fā)現(xiàn)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(nèi)部發(fā)現(xiàn)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, LLC is a privately-held New York-based, biotechnology company focused on discovering and developing treatments for rare and life-threatening diseases. Retrophin is currently developing treatments for Duchenne muscular dystrophy, spinal muscular atrophy, cystic fibrosis and myotubular myopathy. Retrophin's lead internally discovered compound, RE-001, is a protein replacement therapy for Duchenne muscular dystrophy. Retrophin intends to initially develop the drug for rare nephropathies, including Focal Segmental Glomerulosclerosis.