Drug Profile

Research programme: neurodegenerative disorders - Change Cure

Latest Information Update: 30 Apr 2026

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At a glance

Orphan Drug Status

Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

No
New Molecular Entity Yes

Research Amyotrophic lateral sclerosis; Charcot-Marie-Tooth disease; Huntington's disease; Parkinson's disease; Spinocerebellar ataxias

17 Feb 2026 Early research in Amyotrophic lateral sclerosis in Japan (unspecified route), prior to February 2026 (Change Cure pipeline, February 2026)
17 Feb 2026 Early research in Charcot-Marie-Tooth disease in Japan (unspecified route), prior to February 2026 (Change Cure pipeline, February 2026)
17 Feb 2026 Early research in Huntington's disease in Japan (unspecified route), prior to February 2026 (Change Cure pipeline, February 2026)

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