Spinal muscular atrophy workshop
Date:
11/11/2016
Location:
European Medicines Agency, London
A 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.
-
List item
Agenda - EMA - Spinal muscular atrophy workshop (PDF/88.56 KB)
First published: 07/11/2016
Last updated: 07/11/2016 -
List item
Briefing Document to the Clinical Trial Readiness in Spinal Muscular Atrophy (SMA) SMA Europe, TREAT-NMD and European Medicines Agency meeting (PDF/1023.68 KB)
First published: 05/12/2016
Last updated: 05/12/2016 -
List item
Appendix - Briefing Document to the Clinical Trial Readiness in Spinal Muscular Atrophy (SMA) SMA Europe, TREAT-NMD and European Medicines Agency meeting (PDF/155.12 KB)
First published: 05/12/2016
Last updated: 05/12/2016 -
List item
List of participants - Spinal muscular atrophy stakeholder workshop (PDF/130.06 KB)
First published: 05/12/2016
Last updated: 05/12/2016
EMA/790303/2016 -
List item
Presentation - General overview of novel experimental therapeutic approaches in SMA (Francesco Muntoni) (PDF/1.21 MB)
First published: 05/12/2016
Last updated: 05/12/2016 -
List item
Presentation - Presentation - Biomarkers (Arthur Burghes) (PDF/905.11 KB)
First published: 05/12/2016
Last updated: 05/12/2016 -
List item
Presentation - Session 2 discussion and questions (Richard Finkel, Eduardo Tizzano) (PDF/64.12 KB)
First published: 05/12/2016
Last updated: 05/12/2016 -
List item
Presentation - Presentation - Industry perspective on drug development for Type 1 SMA (Kathie Bishop) (PDF/38.84 KB)
First published: 05/12/2016
Last updated: 05/12/2016 -
List item
Presentation - Natural history of SMA and impact of standards of care on survival and motor function (Richard Finkel) (PDF/727 KB)
First published: 05/12/2016
Last updated: 05/12/2016 -
List item
Presentation - SMA type I outcome measures (Basil Darras) (PDF/1.44 MB)
First published: 05/12/2016
Last updated: 05/12/2016 -
List item
Presentation - Strategies for drug development in spinal muscular atrophy (SMA) Type 1 - A regulatory perspective (Marion Haberkamp) (PDF/1.17 MB)
First published: 05/12/2016
Last updated: 05/12/2016 -
List item
Presentation - Exploratory outcome measures (including MRI) (Laurent Servais) (PDF/1.74 MB)
First published: 05/12/2016
Last updated: 05/12/2016 -
List item
Presentation - Types 2 and 3 SMA: Industry perspective (Omar Khwaja) (PDF/296.64 KB)
First published: 05/12/2016
Last updated: 05/12/2016 -
List item
Presentation - The evolving natural history of SMA types I and II (Eugenio Mercuri) (PDF/1.77 MB)
First published: 05/12/2016
Last updated: 05/12/2016 -
List item
Presentation - Functional outcome measures for type 2 and 3 Spinal Muscular Atrophy (Anna Mayhew, Jacqueline Montes) (PDF/2.42 MB)
First published: 05/12/2016
Last updated: 05/12/2016 -
List item
Presentation - Patient perspective to clinical benefit (Elena Mazzone) (PDF/1.46 MB)
First published: 05/12/2016
Last updated: 05/12/2016 -
List item
Presentation - Patient perspective to clinical benefit (Kenneth Hobby, Luca Binetti) (PDF/2.87 MB)
First published: 05/12/2016
Last updated: 05/12/2016 -
List item
Presentation - Registries and trial readiness (Hanns Lochmuller) (PDF/1.11 MB)
First published: 05/12/2016
Last updated: 05/12/2016 -
List item
Presentation - Regulatory perspective (Mario Miguel Rosa) (PDF/292.02 KB)
First published: 05/12/2016
Last updated: 05/12/2016 -
List item
Presentation - Standards of care in SMA and implications for clinical trials (Richard Finkel) (PDF/1.09 MB)
First published: 05/12/2016
Last updated: 05/12/2016