美国Retrophin
retrophin,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, 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.