美國Retrophin
retrophin,LLC是總部位于紐約的一家私人持有的生物技術公司,側重于發現和開發為罕見的危及生命的疾病的治療。 retrophin目前正在開發治療杜氏肌營養不良癥,脊髓性肌萎縮癥,囊腫性纖維化和myotubular肌病。 retrophin的鉛內部發現的化合物,RE-001,是杜氏肌營養不良蛋白替代療法。 retrophin打算初步發展為罕見的腎病,局灶節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.