New treatment for Niemann-Pick type C disease

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Summary

The EMA announced a new treatment, Aqneursa, for Niemann-Pick disease type C, which significantly improves neurological signs, symptoms and functioning.

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Diseases / indications

• Niemann-pick Disease Type

Drugs mentioned

• AQNEURSA (LEVACETYLLEUCINE)

Snippet

Aqneursa significantly improves neurological signs, symptoms and functioning