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risdiplam

Granules for oral solution 60 mg

Dossier type
US CTD
Dossier status
Ready to file
Country of origin
United States
GMP approvals
FDA
Manufacturer #17629
This biopharmaceutical company specializes in developing transformative therapies for patients with rare diseases. Their focus is on addressing the root cause of these conditions, aiming to enhance the quality of life for those affected. Key therapeutic areas include metabolism disorders (such as familial chylomicronemia syndrome, familial partial lipodystrophy, and phenylketonuria) and neurology (covering diseases like amyotrophic lateral sclerosis, Duchenne muscular dystrophy, Friedreich ataxia, Huntington's disease, and spinal muscular atrophy). They operate globally with significant presence in regions like North America, the EU, and Asia. Their product portfolio encompasses prescription medications, leveraging cutting-edge technologies such as gene therapy and splicing modulation. The company is committed to rigorous research and development, evidenced by their ongoing clinical trials and pursuit of innovative treatment options. They hold GMP certifications and have secured patents for several of their products.

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