Curators' meeting - October 2012

Curators Meeting 2012

The sixth annual patient registry curator meeting and fifth oversight committee meeting took place from 29-30 September in Istanbul, Turkey and was attended by 67 participants from 21 countries.

Hanns Lochmüller (Newcastle University, UK) kicked off the meeting with an update on the TREAT-NMD Alliance outlining the new governance structure and future plans for the network. As part of this presentation Hanns discussed the upcoming FP7 funded projects that have been facilitated by TREAT-NMD. All of the FP7 funded projects have a broader remit than just neuromuscular diseases, many of which will have a focus on all rare diseases. Volker Straub (Newcastle University, UK) then talked more in-depth about one project in particular - Neuromics - and how registries will be utilised within the project. Domenica Taruscio (Instituto Superiore di Sanità, Italy) was due to present on the results of the EPIRARE survey and the future plans for RD Connect (another FP7 project) and how this will integrate with registries. As Domenica was unfortunately unable to attend the meeting Hanns Lochmüller presented the slides on her behalf.  Holly Peay (DuchenneConnect Registry, USA) discussed the recently published paper from the DuchenneConnect Registry that was published in PLoS Currents Muscular Dystrophy, and outlined how powerful the data held within registries can be. Holly also presented the results of a study carried out in collaboration with Stanley Nelson (UCLA, USA) who analysed the DuchenneConnect Registry data to try to better understand DMD. The last speaker of the session was En Kimura (National Center of Neurology and Psychiatry, Japan) who provided a broad update on the status of research in neuromuscular diseases in Japan, with a particular focus on registries and collaborations with TREAT-NMD.

The second session of the Curators’ meeting was focused on case studies of patient registries for neuromuscular disorders, which heard from Kathrin Gramsch from the CARE-NMD project, presenting results from the DMD patient survey; John Ditton from the US based biotech company Ultragenyx, presenting a novel public-private partnership model for GNE Myopathy; Guillaume Bassez gave an update on the French-Quebec DM Registry and current research into Myotonic Dystrophy in France; with the last case study hearing from Volker Straub who presented on the Global FKRP Registry and upcoming steroid trial for LGMD2I patients.

The TREAT-NMD Global Database Oversight Committee (TGDOC) meeting took place on Sunday 30 September and discussed important questions relating to registry related studies and the regular housekeeping issues along with the vote for a new Chair and Chair-elect to replace Hanns Lochmüller and Christophe Béroud, who have been Co-chairs since 2007. Jan Verschuuren and Hugh Dawkins were nominated to be the new Chair and Chair-elect respectively. A vote was held by email following the meeting with the result being reached only this week. Unanimous approval for both Jan and Hugh means that they will now step into their new roles with the TGDOC with immediate effect and we wish them luck! Also discussed was proposed changes to the Charter (the document that governs the Global Registries and the TGDOC) to bring it into line with the views and comments of the current TGDOC members. The Charter is discussed every year to ensure that it is fit for purpose. Any changes are reviewed and voted on by the TGDOC with a two-thirds majority needed for the vote to stand.

Simon Woods (Newcastle University, UK), head of the Project Ethics Council for TREAT-NMD, concluded the session with an open discussion on two different ethical issues. The ethical issues were not directly related to registries but neuromuscular disorders in general and were very thought provoking.

The next curator and OC meeting will take place in Autumn 2013, venue still to be confirmed.

A full list of all existing national registries for DMD, SMA and Myotonic Dystrophy can be found here.

12 Apr 2017