Spinal muscular atrophy workshop

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Date: Friday, 11 November 2016, All day
Location: European Medicines Agency, Amsterdam, the Netherlands

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.

Documents

Agenda - EMA - Spinal muscular atrophy workshop

English (EN) (88.56 KB - PDF)

First published: 07/11/2016 Last updated: 07/11/2016

Briefing Document to the Clinical Trial Readiness in Spinal Muscular Atrophy (SMA) SMA Europe, TREAT-NMD and European Medicines Agency meeting

English (EN) (1023.68 KB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

Appendix - Briefing Document to the Clinical Trial Readiness in Spinal Muscular Atrophy (SMA) SMA Europe, TREAT-NMD and European Medicines Agency meeting

English (EN) (155.12 KB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

List of participants - Spinal muscular atrophy stakeholder workshop

Reference Number: EMA/790303/2016

English (EN) (130.06 KB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

Presentation - General overview of novel experimental therapeutic approaches in SMA (Francesco Muntoni)

English (EN) (1.21 MB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

Presentation - Presentation - Biomarkers (Arthur Burghes)

English (EN) (905.11 KB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

Presentation - Session 2 discussion and questions (Richard Finkel, Eduardo Tizzano)

English (EN) (64.12 KB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

Presentation - Presentation - Industry perspective on drug development for Type 1 SMA (Kathie Bishop)

English (EN) (38.84 KB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

Presentation - Natural history of SMA and impact of standards of care on survival and motor function (Richard Finkel)

English (EN) (727 KB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

Presentation - SMA type I outcome measures (Basil Darras)

English (EN) (1.44 MB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

Presentation - Strategies for drug development in spinal muscular atrophy (SMA) Type 1 - A regulatory perspective (Marion Haberkamp)

English (EN) (1.17 MB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

Presentation - Exploratory outcome measures (including MRI) (Laurent Servais)

English (EN) (1.74 MB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

Presentation - Types 2 and 3 SMA: Industry perspective (Omar Khwaja)

English (EN) (296.64 KB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

Presentation - The evolving natural history of SMA types I and II (Eugenio Mercuri)

English (EN) (1.77 MB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

Presentation - Functional outcome measures for type 2 and 3 Spinal Muscular Atrophy (Anna Mayhew, Jacqueline Montes)

English (EN) (2.42 MB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

Presentation - Patient perspective to clinical benefit (Elena Mazzone)

English (EN) (1.46 MB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

Presentation - Patient perspective to clinical benefit (Kenneth Hobby, Luca Binetti)

English (EN) (2.87 MB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

Presentation - Registries and trial readiness (Hanns Lochmuller)

English (EN) (1.11 MB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

Presentation - Regulatory perspective (Mario Miguel Rosa)

English (EN) (292.02 KB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

Presentation - Standards of care in SMA and implications for clinical trials (Richard Finkel)

English (EN) (1.09 MB - PDF)

First published: 05/12/2016 Last updated: 05/12/2016

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