Spinal muscular atrophy workshop

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TitleSpinal muscular atrophy workshop
Date11/11/2016 - 11/11/2016
LocationEuropean Medicines Agency, London
SummaryA one-day workshop on spinal muscular atrophy (SMA) co-organised by the European Medicines Agency, SMA Europe and TREAT NMD. The workshop brings together key stakeholders to discuss, help and advance the development of therapies for the treatment of SMA. Topics for discussion include an overview of the disease, the pharmacology of the molecules under investigation, natural history data, clinical outcome measures and biomarkers.

All documents

Name Language First published Last updated
Agenda - EMA - Spinal muscular atrophy workshop (English only) 2016-11-07  
Briefing Document to the Clinical Trial Readiness in Spinal Muscular Atrophy (SMA) SMA Europe, TREAT-NMD and European Medicines Agency meeting (English only) 2016-12-05  
Appendix - Briefing Document to the Clinical Trial Readiness in Spinal Muscular Atrophy (SMA) SMA Europe, TREAT-NMD and European Medicines Agency meeting (English only) 2016-12-05  
List of participants - Spinal muscular atrophy stakeholder workshop (English only) 2016-12-05  
Presentation - General overview of novel experimental therapeutic approaches in SMA (Francesco Muntoni) (English only) 2016-12-05  
Presentation - Presentation - Biomarkers (Arthur Burghes) (English only) 2016-12-05  
Presentation - Session 2 discussion and questions (Richard Finkel, Eduardo Tizzano) (English only) 2016-12-05  
Presentation - Presentation - Industry perspective on drug development for Type 1 SMA (Kathie Bishop) (English only) 2016-12-05  
Presentation - Natural history of SMA and impact of standards of care on survival and motor function (Richard Finkel) (English only) 2016-12-05  
Presentation - SMA type I outcome measures (Basil Darras) (English only) 2016-12-05  
Presentation - Strategies for drug development in spinal muscular atrophy (SMA) Type 1 - A regulatory perspective (Marion Haberkamp) (English only) 2016-12-05  
Presentation - Exploratory outcome measures (including MRI) (Laurent Servais) (English only) 2016-12-05  
Presentation - Types 2 and 3 SMA: Industry perspective (Omar Khwaja) (English only) 2016-12-05  
Presentation - The evolving natural history of SMA types I and II (Eugenio Mercuri) (English only) 2016-12-05  
Presentation - Functional outcome measures for type 2 and 3 Spinal Muscular Atrophy (Anna Mayhew, Jacqueline Montes) (English only) 2016-12-05  
Presentation - Patient perspective to clinical benefit (Elena Mazzone) (English only) 2016-12-05  
Presentation - Patient perspective to clinical benefit (Kenneth Hobby, Luca Binetti) (English only) 2016-12-05  
Presentation - Registries and trial readiness (Hanns Lochmuller) (English only) 2016-12-05  
Presentation - Regulatory perspective (Mario Miguel Rosa) (English only) 2016-12-05  
Presentation - Standards of care in SMA and implications for clinical trials (Richard Finkel) (English only) 2016-12-05  

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