Contemporary outcome measures in neuromuscular diseases

Concept
Improving clinical outcomes remains the gold standard in advancing healthcare. The only way to successfully manage care is to measure it. Outcome measurement is used, on the one hand, in clinical practice and in the other hand, their use in the clinical trials aims to demonstrate the effect of a treatment and/or therapeutic technique from a clinical point of view in order to justify the use of this treatment. For these reasons, the overall objective of the workshop is to focus on all existing outcome measures used in neuromuscular diseases. lt will consist of both presentations by experts in the field of neuromuscular diseases as well as interactive panel discussions and quiz sessions to train participants in performing and interpreting these outcome measures.

Aim
In recent years, digital biomarker development has begun integration into translational and clinical research. An increasing number of industry and academic investigators are at the leading edge of a new wave of innovations. This workshop aims to deliver participants a training focusing an innovative aspects by presenting the advancement of technologies with digital outcome measures. Participants will be trained in how to make the methodology evolve and how digital biomarkers could be considered as a complementary approach to current approaches for outcome capture. Digital biomarkers allow to directly collect the data about health or disease management through digital health technologies to explain influence, and/or predict health-related results. They span a broad range of diagnostic and prognostic measurements that are frequently made outside of the clinical environment using home-based connected products including wearable, implantable, and ingestible devices and sensors. They offer the possibility to overcome many of the limitations of the traditional outcome measures and offer unique advantages like scale, cost, capture frequency, data volume, measurement objectivity, setting captured, and engage patients. This workshop also delivers a cross-cutting training: the digital biomarkers have wide range of applications in healthcare, with devices built for dermatology, nephrology, inflammatory diseases, metabolic diseases, cardiology, neuromuscular diseases.

The workshop is open for different target groups, especially those who want to deepen their knowledge in clinical outcomes measures and innovative digital outcomes as researchers and clinicians affiliated to an ERN-Full Member or Affiliated Partner Institution, physiotherapists and students.

On site: 20 participants
Videoconference: 50 participants

The workshop will provide a proactive top-quality learning training on the ways in which outcomes are captured and will train participants on the different scales. This existing assessment scales in neuromuscular diseases identification will aim participants to discuss their relevance and limitations. At the end of each session, there will be Multiple Choice Questions (MCQ) including practical situations and interactive panel discussions.

To register please fill the application form here. Registrations are still possible until the end of February but only for online participation in the workshop.

The training workshop is free of charge.

Affiliated to an ERN-Full Member or Affiliated Partner institution.

The organizer together with one member of the WP17 Scientific Evaluation Committee will select the most appropriate workshop participants based on their expertise in the field of outcome measures in neuromuscular diseases.

All registered participants will be informed if they are selected to attend this training course.

At the end of the training workshop a Certificate of Attendance will be sent to the participants who attended the entire workshop.

Workshops will run in English.

Hotel Novotel Nice Centre Vieux-Nice
8-10 Esplanade du Parvis de l’Europe
06300 NICE
France (On site and by videoconference).

If you have specific questions, please write to the workshop organizer:
avari.s@chu-nice.fr

Questions related to organization of travelling hotel, dinner should be addressed to:
alexine@abc-collective.com

For general inquiries about the workshops in the ERN RD Support and Training Program please write to:
Sanja.hermanns@ejprd-project.eu
RareEndoERN@Iumc.n1

You will find the programme here.