Leonard van den Berg UMC Utrecht, the Netherlands

Buildingbridgesandbreakingbarriersinrareneuromusculardiseases 2

Specialisedgroup:MotorNeuronDiseases43HCPs

Buildingbridgesandbreakingbarriersinrareneuromusculardiseases 3

Motor Neuron Diseases Specialist Group

Leonard van den Berg (chair) Ludo van der Pol (vice-chair) Carlos Vrins (secretary)

Evy Reviers (ALS) Mencía de Lemus Belmonte (SMA)

ALS subgroup ± 26 HCPs

SMA subgroup ± 30 HCPs

Coordinators PatientRepresentatives

Buildingbridgesandbreakingbarriersinrareneuromusculardiseases

•  Structure–  Board– Meetings–  Existingconsortia

•  Prioritise–  Guidelines–  Trial(readiness)–  Education

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Motor Neuron Diseases Specialist Group

Buildingbridgesandbreakingbarriersinrareneuromusculardiseases

SubgroupALSLeonardvandenBerg

•  ERN ENCALSèTRICALS

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Buildingbridgesandbreakingbarriersinrareneuromusculardiseases

SubgroupSMALudovanderPol

•  Standardsofcare– AttentionforSOCforadults

•  Standardsoftreatment– Differencesbetweencountries(spinrazareimbursement)

•  Emergingtherapies– Registries:connect&standardise

•  TREAT-NMDworkshop,8-9may2017(report)

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