International Niemann–Pick Disease Alliance

AIDNPC – summary of teleconference minutes 31st March 2016

A teleconference was hosted by Orphazyme on 31st March 2016, to provide an update to patient organisations regarding the development of Arimoclomol in the AIDNPC programme. The next call is scheduled to take place 28th April 2016.

In summary, the call covered:

  • 001 Study: Recruitment progressing well — 30 patients enrolled at 11 sites
  • 002 Study: Two objective motor function endpoints added to protocol
  • 002 Study: Filing of IND in the US and of CTAs in Europe
  • Review: Arimoclomol in treatment of sIBM patients

Orphazyme provided the following summary AIDNPC Update #4.

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