International Niemann–Pick Disease Alliance
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: