International Niemann–Pick Disease Alliance

Orphazyme announces update to its AIDNPC clinical programme

Orphazyme has announced a further update to its NP-C clinical programme.

Arimoclomol will require the performance of a double-blinded clinical trial with a control group.

The control group means that a third of patients enrolled will receive a placebo for a period of 12 months. After 12 months, all patients will be offered Arimoclomol in an open label extension.

For more information, see their website:

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