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DMD and Gamified Physiotherapy
Duchenne Muscular Dystrophy (DMD) is a progressive neuromuscular disorder that leads to loss of muscle strength, functional decline, reduced mobility, and limitations in participation. Early and ongoing physiotherapy is essential to slow functional deterioration, maintain mobility, and support participation in daily-life activities. Despite its importance, adherence to long-term exercise programs is frequently low among children with DMD due to fatigue, monotony, lack of feedback, limited motivation, and challenges related to time management and caregiver burden.
Gamification has emerged as a promising method to sustain motivation and engagement by integrating game-based elements such as challenges, levels, rewards, feedback, and achievement-based progression into non-game health contexts. Digital and mobile health solutions also provide an opportunity to deliver personalised and home-based rehabilitation, support remote follow-up, and increase continuity of care. However, evidence for gamified telerehabilitation specifically designed for DMD is limited, and most existing telerehabilitation programs lack systematic motivational components or long-term follow-up data.
This study evaluates a gamified mobile physiotherapy exercise program developed for children with DMD to increase motivation, participation, and adherence to home-based rehabilitation. The program includes personalised exercise tasks designed by physiotherapists and occupational therapists, and integrates gamification mechanisms to reinforce engagement. The intervention is delivered alongside an 8-week center-based physiotherapy program and followed for an additional 6 months to assess long-term adherence and carry-over effects.
A total of 46 boys aged 6-12 years with a confirmed diagnosis of DMD will be recruited and randomly assigned to an intervention group or a control group. Both groups receive an identical center-based physiotherapy program, whereas the intervention group additionally uses the mobile application at home. The control group receives standard home-exercise instructions. Assessments occur at baseline, post-intervention, and at a 6-month follow-up.
Primary outcomes focus on physical function, mobility, and endurance. Secondary outcomes include psychosocial well-being, motivation, and therapy participation. This study aims to determine whether a gamified telerehabilitation approach can enhance adherence, help preserve physical function, and support participation in children with DMD. If effective, this digital model may offer an accessible, motivating, and scalable strategy for long-term rehabilitation in pediatric neuromuscular disorders.
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A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD)
Actively Recruiting
2+ Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:Restoring or Replacing Dystrophin
Variant Requirement:Duchenne - variant criteria varies by cohort
A Study to Investigate the Safety and Biodistribution of a Single Intrathecal (IT) Injection of INS1201 in Ambulatory Males With Duchenne Muscular Dystrophy (DMD)
Actively Recruiting
2 Years to <5 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:Restoring or Replacing Dystrophin
Variant Requirement:Duchenne - variants in exons 18 to 58
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO)
Actively Recruiting
4 Years to <19 Years
Restoring or Replacing Dystrophin
Actively Recruiting
Therapeutic Approach:Restoring or Replacing Dystrophin
Variant Requirement:Duchenne - amenable to exon 51 skipping
A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
Active, Not Recruiting
8 Years to 18+ Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:Restoring or Replacing Dystrophin
Variant Requirement:Duchenne - Excluding any deletion of exon 8 and/or 9
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
Active, Not Recruiting
4 Years to <17 Years
Restoring or Replacing Dystrophin
Active, Not Recruiting
Therapeutic Approach:Restoring or Replacing Dystrophin
Variant Requirement:Duchenne - amenable to exon 51 skipping