Revised standards of care were recently published in Neuromuscular Disorders (part 1 & part 2). Coordinated by Treat-NMD, SMA Europe has worked with members of other organisations, on a version for families.
A workshop on SMA standards of care, supported by the ENMC, together with SMA Europe, SMA Support UK (now SMA UK), Cure SMA and the SMA Foundation, was held. This initiative gathered twenty-one clinical researchers from seven different countries (USA , UK , Italy , Germany , France , Sweden , Denmark ), 2 ENMC representatives and 4 representatives of patient advocacy groups (SMA Europe: board member Anna Witchen and vice-President Mencia de Lemus), SMA Foundation , Cure SMA ).
The aim of the workshop was to review and update the 2007 standard of guidelines for spinal muscular atrophy. The workshop was conducted under the leadership of Richard Finkel, Eugenio Mercuri and Thomas Sejersen. This workshop focused upon 8 topics, each with a working group of 2 or 3 co-leaders who then selected approximately 10 international experts on that topic to participate in a structured review and discussion. Detailed literature review of available information preceded the working group’s expert opinion process, which utilised the Delphi technique, a structured sequential analytic process, to arrive at a group consensus. Input from patient representatives and several pharmaceutical companies working on drug development for SMA was sought.