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Personalized Training for People With Rare Neuromuscular Disorders

Personalized Training for People With Rare Neuromuscular Disorders

Recruiting
18-70 years
All
Phase N/A

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Overview

The goal of this study is to investigate the effects of personalized exercise treatment on dynamic balance and physical function in comparison with regular follow-up in adults with rare-neuromuscular disorders: Charcot-Marie-Tooth (CMT), Facioscapulohumeral Muscular Dystrophy (FSHD), and Myotonic Dystrophy Type 1 (DM1).

The key objectives are:

  1. To investigate if the intervention group experiences improvements in dynamic balance that are superior to the control group
  2. To investigate if the intervention group experiences long-term improvements in dynamic balance that are superior to the control group during the follow-up
  3. To investigate if improvements in dynamic balance are associated with improvements in physical activity, body composition, estimated motor units, metabolomics, muscle echnogenecity and volume, and other indicators of health and quality of life.

This is a national study and will involve 120 individuals with rare-neuromuscular disorders from Norway's four health regions.

Eligibility

Inclusion Criteria:

  • A confirmed diagnosis of either FSHD, DM1 or CMT
  • 18-70 years of age at the time of signing the informed consent.
  • Any gender
  • Ability to stand, rise from a chair and walk at least 10 meters with or without any need of assistive devices
  • Indication for rehabilitation as confirmed by the treating neurologist or physiotherapist
  • Ability to understand and follow instructions in Norwegian
  • Capable of giving signed informed consent

Exclusion Criteria:

  • Pregnancy or planning to become pregnant
  • Any other neurological or non-neurological disorders affecting physical capacity, such as disabling arthritis, severe heart-failure/cardiomyopathy, on-going cancer treatment
  • Alcohol or drug abuse as per their medical chart
  • History of non-compliance to medical advice/follow-up

Study details
    Neuromuscular Diseases (NMD)
    Charcot Marie Tooth Disease (CMT)
    Facioscapulohumeral Muscular Dystrophy
    Myotonic Dystrophy Type 1 (DM1)

NCT06708468

Oslo University Hospital

16 October 2025

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