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30th November 2011
Patient registry curators from across the world come together in Geneva

The fifth annual patient registry curator meeting and fourth oversight committee meeting, which took place from November 11-12 in Geneva, Switzerland, was attended by 68 participants from 33 countries. Attendees heard a variety of presentations on topics from next-generation sequencing to patient care evaluation, and case studies were presented outlining the usefulness of the patient registries in clinical trial planning and recruitment, including the successful recruitment of patients through the registries for two separate trials.

The TREAT-NMD Global Database Oversight Committee (OC) also met to discuss important questions relating to the way patients are recruited for clinical trials through the patient registries, a discussion which raised thought-provoking ethical discussions on cross-border participation in clinical trials, as well as the issue of what happens to patients when trials are discontinued or cancelled, and how to deal with competing trials recruiting the same patient cohort. Recognition of the utility of the global registries comes from all stakeholders: during the public consultation (October 2010, 430 respondents from all stakeholder groups) the patient registries came out as the second highest priority, with only the broader "facilitation of collaboration" ahead of it as the top priority. All of the curators and OC members agreed that the collaboration and patient registries should be sustained as TREAT-NMD moves beyond its current EU funding period and develops into the TREAT-NMD Alliance.

25th French Telethon - meet the challenge!
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The 25th Telethon will take place on 2nd and 3rd December. Since 1987, this annual event - initiated by the French muscular dystrophy association AFM - has been an exceptional example of solidarity and generosity that has given hope and support to individuals with neuromuscular and other rare diseases not only in France but across the world. Collecting almost 100 million Euros per year, the Telethon supports numerous research programmes and clinical trials  - currently funding 36 trials in 31 rare diseases - and enables provision of information services and socially innovative support services. The 25 years of the Telethon have seen revolutions in genetics, biotherapies, and social care. Major scientific advances have been made, and today the first treatments are emerging. This year's Telethon will take place across six French public TV channels and last more than 30 hours. Across France, members of the public are signing up with their own specific "challenges" to raise money - from shaving off their beards to combating fear of snakes - and the TV marathon will be a fun tribute to the fundraising work of the countless supporters, the stories of those affected by NMDs, and the doctors and scientists working towards therapies.

Read more by clicking the "more" link below, or make a donation here.

European Conference on Rare Diseases and
Orphan Products 2012 Update
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The European Conference on Rare Diseases and Orphan Products provides a unique platform covering all rare diseases, across all European nations. With over 100 speakers and countless professionals in attendance, this annual conference covers research and therapy development and information regarding health care, social care, public health issues and support at both the European and national levels.

Early bird registration is currently underway and a call for poster abstracts is open on the conference website.

The poster abstract themes are: National Plans for Rare Diseases, Centres of Expertise (CoE) & European Reference Networks (ERN), Information & Public Health, Research from Bench to Bedside, Orphan Products & Rare Disease Therapies, Patients’ Empowerment, Rare Disease Patient Groups Innovations, and an open topic.

The closing date for abstract submission is 15th January 2012.

Download the conference flyer or visit the conference website for further details of the programme, speakers and venue.

LGMD2I Research Fund launches website
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The LGMD2I Research Fund has just launched its website: www.lgmd2ifund.org. This non-profit private foundation, established in March 2011, focuses on finding treatments for Limb Girdle Muscular Dystrophy 2I and other alpha-dystroglycanopathies. Through its grants awards program, the LGMD2I Research Fund seeks to foster basic research in the field, to promote clinical trial readiness and to support translation of promising scientific discoveries into clinics. Visit the LGMD2I Research Fund website for more information on its newly awarded grants or on resources for patients and the scientific community.

Myotonic Dystrophy Family Conference to be streamed live online
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In conjunction with IDMC-8, the eighth Myotonic Dystrophy Consortium congress, the Myotonic Dystrophy Foundation's EMPOWER 2011: Myotonic Dystrophy Family Conference is about to take place. This coming weekend, 400 people from seven countries and 36 US states will attend the 2nd annual conference in Clearwater Beach, Florida, USA. Many of the sessions will be filmed and subsequently made available online. In addition, the MDA is providing a video live stream of the joint session of IDMC-8 and the MDF's Family Conference on Saturday, December 3, 2011 from 2pm to 6pm ET (11am to 2pm PT).

Visit www.mda.org/IDMC8 on the day to see these sessions live online.

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30th November 2011
TREAT-NMD newsletter - 30th November 2011
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