Curators' meeting - September 2009

Third curator training meeting shows TREAT-NMD patient registries are going from strength to strength!

Curator meeting 2009

DMD & SMA registriesMore than 90 participants from 23 countries attended the third annual patient registry curator training meeting, which took place from September 7–9 in Montpellier, France. Over 30 countries worldwide are now part of the TREAT‐NMD global registries initiative for Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA) and have national registries either already established and enrolling patients or in the set up phase. Participants at the meeting received training in correct annotation of the genetic diagnosis and discussed the registry dataset. The Global Database Oversight Committee, the body responsible for the ethical governance of the registry, also met to discuss important questions relating to the way patients are recruited for trials through the registries.

DMD and SMA registries: coverage going global

The TREAT‐NMD patient registries were set up primarily with future trials and therapies in mind. The global DMD and SMA registries are recognised as the leading resource for trial planning and recruitment in these diseases at an international level and are already being used by pharmaceutical companies for this purpose. They function on the basis of multiple national systems feeding into a single international database. The national registries all collect the same streamlined, internationally harmonised core dataset that includes key information facilitating trial planning and enabling patient recruitment. There is a strong ethos of patient involvement in the project, with many of the registries run by patient organizations and all registries committed to providing regular feedback to patients about new information relevant to their condition, as well as potential trials or new treatment. "We are thrilled to see how many countries are now part of this global initiative," commented Hanns Lochmüller, leader of the registries initiative for TREAT‐NMD. "It's a real testament to the hard work of all our colleagues worldwide, both patients and specialists, and we are immensely grateful to all of them for their support. Everyone present at this training meeting had a real sense of the power of this initiative and could feel part of a global network." "The primary purpose of the registries is to help patients get recruited into trials for new treatments, and patients themselves want this as much as the companies who are now coming to us for data," added global registry coordinator Christophe Béroud. "But equally important is to be able to feed back to patients worldwide about all the important developments relating to their condition, and to help them feel more closely linked to the research community. This meeting showed that both these aims are becoming a reality."

A full list of all the existing national registries for DMD and SMA can be found at www.treat‐

Registries for other conditions: broadening the initiative

For the first time, representatives of registries for other neuromuscular diseases were also present, and participants heard about the plans for an international registry for patients with myotonic dystrophy type 1 (DM1), plus the recent launch of a congenital muscular dystrophy registry, the upcoming launch of a registry for patients with FKRP mutations (LGMD2I and MDC1C) and others. All these registries share the same aims of locating patients in preparation for future trials, and providing feedback and a sense of community to the patients affected by these conditions.

12 Apr 2017