Patient Registries and Trial Readiness in Myotonic Dystrophy--TREAT-NMD/Marigold International Workshop Report.

摘要

A workshop entitled Patient Registries and Trial Readiness in Myotonic Dystrophy, jointly sponsored by TREAT-NMD (www.treat-nmd.eu) and the Marigold Foundation (www. marigoldfoundation.org), was held from 12 to 14 June 2009 in Naarden, The Netherlands. The twenty-six participants represented eight countries and included scientists, clinicians, patient representatives and industry. The workshop built on the foundations established in two previous ENMC workshops on myotonic dystrophy and the myotonic dystrophy clinical working group set up by the Marigold Foundation, and took advantage of the tools developed within the TREAT-NMD network for patient registries and outcome measures.Opening remarks from the workshop organizers, Hanns Loch-muller (TREAT-NMD) and Karla Blonsky (Marigold Foundation), showed that in recent years the need to collect patient data in a harmonized manner across multiple countries has become increasingly evident in the rare disease field, where locating patients suitable for a particular trial or therapy poses a particular challenge. Baziel van Engelen summarized previous ENMC (European Neuro-muscular Centre) workshops on DM1 [1 ] which identified the need for international collaborations on outcome measures, natural history, patient registries and clinical trials. The present workshop also drew on the conclusions of the 157th ENMC International Workshop: Patient registries for rare, inherited muscular disorders, 25-27 January 2008 [2].