Clinical Research Directory

High-Tech Rehabilitation Pathway for Acute Adult Neuromuscular Diseases - Fit4MedRob-Acute MND Project

The goal of this study is determine if a high-tech rehabilitation circuit is more effective than usual rehabilitation methods in improving functional outcome, particularly balance control, for patients with acute neuromuscolar diseases. The main question it aims to answer is: Are high-tech rehabilitation interventions, including robotic systems, virtual reality, and stabilometric platforms, more effective than traditional rehabilitation methods in improving balance, motor function, fatigue levels, sarcopenia, cognitive engagement, and overall quality of life in patients with acute neuromuscular diseases (NMDs)? Researchers will compare a robotic treatment group, that consists in an high-tech rehabilitation, with a control group, that will receive the traditional rehabilitative treatment.

Our Study Aims to Determine if Nerve Alterations in Acute GBS and CIDP Detectable by Ultrasound Match Electrodiagnostic Findings and if This Method Aids Early Diagnosis, Predict Their Outcomes and Differentiate Between Axonal and Demyelinating Subtypes.

Neuromuscular ultrasound (NMUS) is emerging as a valuable non-invasive diagnostic tool. In GBS, NMUS can detect proximal nerve enlargement early, before neurophysiological changes. Persistent nerve enlargement can be observed up to 15 years, though its correlation with disability varies. Research is needed to clarify NMUS findings in GBS and CIDP over time. Early detection of nerve root enlargement via NMUS could facilitate earlier diagnosis and intervention, improving patient outcomes and understanding of these conditions\' pathophysiology. This study aims to determine if nerve alterations in acute GBS and CIDP detectable by ultrasound match electrodiagnostic findings and if this method aids early diagnosis. The investigators will perform serial nerve ultrasounds and NCS to investigate nerve morphology, predict outcomes, and differentiate between axonal and demyelinating subtypes.

Living With Guillain-Barré Syndrome as Children.

The goal of this qualitative observational study is to explore the lived experience and psychological aftermath of children who were affected by Guillain-Barré Syndrome (GBS) two to five years earlier. GBS is a rare and acute neurological condition, and while motor recovery is generally good in children, residual symptoms such as fatigue, pain, anxiety, or depression may persist and impact quality of life. The main questions it aims to answer are: How do children experience and recall their illness and recovery after GBS? What psychological or contextual factors might contribute to emotional distress or depressive symptoms in the years following the disease? Participants will: Be children aged 10 or older who had GBS two to five years prior. Participate in a 45-60-minute semi-structured individual interview during a routine follow-up visit. Respond to a screening questionnaire for depressive symptoms (Children's Depression Inventory or Beck Depression Inventory Fast Screen). Undergo a brief clinical examination to assess any remaining physical sequelae. Interviews will be audio recorded, transcribed verbatim, and pseudonymized. Thematic reflexive analysis will be performed following Braun and Clarke's methodology. Recruitment will continue until theoretical saturation is reached (approximately 25 participants expected) across two centers (Toulouse and Montpellier). This study aims to generate new insights into pediatric psychological outcomes after GBS, in order to improve acute care and follow-up, and potentially guide future mental health support for affected children.

The Use of Assistive Gait Devices Can Reduce the Risk of Falls in Patients With Neuromuscular Diseases Following a Training Period.

The planned project is an intervention study to assess the risk of falling after adaptation of an assistive gait devices in patients with the following neuromuscular diseases: Inclusion body myositis, myotonic dystrophy, limb girdle and facioscapulohumeral muscular dystrophies, Pompe disease, Lambert-Eaton syndrome, myasthenia gravis, spinal muscular atrophy, Guillain-Barré syndrome, chronic inflammatory demyelinating polyradiculoneuropathy, Friedreich's ataxia and hereditary motor and sensory neuropathy. The primary aim is to assess the risk of falling after a suitable assistive gait device has been provided with an adaptation phase through training. The data should help to improve the provision of aids for patients with neuromuscular diseases. This should have a positive effect on the risk of falling and thus improve quality of life and reduce mortality and morbidity. To achieve these goals, a one-week intervention with training sessions on handling, balance and coordination as well as fall prevention will becarried out after the patient has been fitted with a suitable assistive gait device. The interventions will be embedded in the inpatient rehabilitation programme. The functional gait and balance tests 'Timed Up and Go', '10 metre walk test', '6-minute walk test' and 'Dynamic Gait Index' will be recorded additionally. The Falls Efficacy Scale International questionnaire will be utilised to evaluate the risk of falling, while the Quebec User Evaluation of Satisfaction with Assistive Technology questionnaire will be employed to ascertain satisfaction with the assistive devices. The study is scheduled to run for a period of 14 days, during which participants will undergo three functional walking and balance tests. As part of the inpatient rehabilitation programme, participants will undergo a week-long period of rehabilitation without assistive technology, followed by a subsequent week of rehabilitation with adapted assistive technology.

Upper Limb Robot-Assisted Therapy in Patients with Guillain-Barré Syndrome

Guillain-Barré Syndrome is an immune-mediated polyradiculoneuropathy, with around 100,000 new cases reported worldwide annually. The aim of this double-blind randomized controlled trial with a SHAM control group is to evaluate the effects of robot-assisted therapy, integrated with conventional therapy, on upper limb motor recovery in patients with GBS.

Neuromuscular Complications of MEK Inhibitors: a French Case Series and a Systematic Review of the Literature

MEK inhibitors (trametinib, cobimetinib, selumetinib or binimetinib) have been used since 2016 to treat metastatic melanoma, by targeting the MAPK pathway Neuromuscular complications (neuropathy, myasthenia or myositis) have been reported in patients treated with MEK inhibitors. With the growing use of these new oncology therapies, neurologists, oncologists and other clinicians are likely to be increasingly confronted with MEK inhibitor-induced neuropathy, myasthenia or myositis. Yet, so far, these complications have only been documented in a few single case reports. Our aim was to characterize the neuromuscular complications associated with MEK inhibitors used either alone or in combination.