Experimental Rett Therapy Now an Orphan Drug in Europe

Experimental Rett Therapy Now an Orphan Drug in Europe

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Experimental Rett Therapy Now an Orphan Drug in Europe

The European Commission has granted orphan drug designation to TSHA-102, an investigational gene therapy for the treatment of Rett syndrome. The designation encourages the development of medicines to diagnose, prevent, or treat life-threatening or debilitating diseases that affect fewer than five in 10,000 people living in the European Union. Its benefits extend protocol assistance in clinical trials, reduced regulatory fees and, if approved, market exclusivity. “The receipt of orphan drug designation from the European Commission…

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