Outcome measures and clinical trial readiness in primary mitochondrial myopathies in children and adults – ERN-EURO-NMD

About EURO-NMD – & ERNs
Patient – engagement
Educational – resources
Guidelines – & protocols
Research – & clinical trials
Annual Meeting – 7-8 Nov
.
- Close
.
- Get in touch
  
  Please note: We can’t respond to individual patient cases from this form. Do not share any patient information through this email system.
  
  We will only use the information you send to us via this form to deal with your particular question. We will not add any details left here to our mailing lists and it will not be shown on our website.
  
  Once 'we' have been in touch with you we will delete your details from the system.
  
  'We' means the EURO-NMD Secretariat who are in overall charge of the website and its content.
  
  Are you happy with us storing the details below whilst we deal with your enquiry? (required)
  
  Yes, I'm happy for you to keep my data for this purpose
  
  Your name(required)
  
  Your email address(required)
  
  Subject(required)
  
  Your message(required)
  
  Are you human?(required)
  
  Please note: We can’t respond to individual patient cases or information sent to us via this form.
  
  This field should be left blank
  
  Please wait...
- Close

Outcome measures and clinical trial readiness in primary mitochondrial myopathies in children and adults. Consensus recommendations

Type of publication: Guidelines, Outcome measures, Workshop report
Disease: Mitochondrial disease

Authors:

Mancuso M, McFarland R, Klopstock T, Hirano M; consortium on Trial Readiness in Mitochondrial Myopathies.

Twenty-six researchers from 10 different countries (USA, Spain, Italy, France, Germany, The Netherlands, United Kingdom, Japan, Norway and Canada) met in Rome, Italy, from 16–18 November 2016 to update current knowledge on clinical trial readiness and outcome measures for Primary Mitochondrial Myopathies (PMM). Patients’ advocacy groups delegates also attended.

Published: 12 December 2017
Journal: Neuromuscular Disorders, volume 27, issue 12, pages 1126-1137

Link: doi.org/doi.org/10.1016/j.nmd.2017.08.006

This publication is recommended by the Mitochondrial Diseases Working Group group.

The ERNs are co-funded by the
European Union (Health Programme and CEF)

“EURO-NMD is one of the 24 European Reference Networks (ERNs) approved by the ERN Board of Member States. The ERNs are co-funded by the European Union (Health Programme and CEF).
For more information about the ERNs and the EU health strategy,
please visit ec.europa.eu/health/ern“