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Sep 20, 2023 471 likes •568 views

Making sense a future perspective Making outcomes work SCOPE-DMD Workshop Annemieke Aartsma-Rus London, May 26 2016 COST Action BM1207 core group meeting 2 Department of Human Genetics Annemieke Aartsma-Rus Meeting April 29

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Making sense – a future perspective “Making outcomes work” – SCOPE-DMD Workshop

Annemieke Aartsma-Rus

London, May 26 2016

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Annemieke Aartsma-Rus Department of Human Genetics 2

COST Action BM1207 core group meeting

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Annemieke Aartsma-Rus Department of Human Genetics 3

Meeting April 29 2016

http://exonskipping.eu/media/videos-workshop-2015/

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Annemieke Aartsma-Rus Department of Human Genetics 4

Paper will be online June 6

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Annemieke Aartsma-Rus Department of Human Genetics 5

Take home messages

Need a collaborative and constructive dialogue

between all stakeholders

Need to develop and implement care standards
Involve all stakeholders to develop functional

and molecular outcome measures

Collect high-quality data for natural history and
utcome measures
Trial design
Regulatory approval

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Annemieke Aartsma-Rus Department of Human Genetics 6

Future directions (focus on OMs)

Define a core set of outcome measures for clinical

trials

Develop outcome measures for non-ambulant

patients

Involve regulators in development of outcomes

measures

Publish date on natural history and outcome

measures è This meeting

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Annemieke Aartsma-Rus Department of Human Genetics 7

Wrap up – Molecular biomarkers

DNA and serum biomarkers
Standardization and collaboration
MRI
Correlation with function and strength
Many trials now include MRI
Regulatory requirements
Efficacy measurements
Data and quality important (collect, collaborate and share!)
Training important (standardization/replication)
Secondary outcomes from patients important (PROM & QoL)
Come early and discuss – regulators are willing to help
Choose appropriate marker(s) (therapy and stage)
Involve patients

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Annemieke Aartsma-Rus Department of Human Genetics 8

Wrap up – Functional outcomes

Once scale cannot measure everything for all stages
Upper limb
Myotools, activity monitors, PUL, reachable workspace
Relation strength and function and quality of life
Decrease in upper limb function takes decades
Selection of outcome measures (stage related)
Collaboration with patients
Combined efforts: PUL and PROM (DMD-Upper PROM)
Collaboration with patients to develop scales and trial design

(drug development starts and ends with patients)

Meaningful
Regulators need data confirming correlation to function that

is important to patient

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Annemieke Aartsma-Rus Department of Human Genetics 9