Duchenne Muscular Dystrophy research hub
13 indexed studies · 11 currently recruiting
- recruitingPHASE2
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients
This study is testing a new medicine, SAT-3247, for boys aged 7-9 with Duchenne muscular dystrophy. We want to find the best dose, see if it's safe, and check if it helps. Some boys will get the real medicine, others a dummy medicine.
United States · Australia · Belgium - recruiting
Can Waveform and Flow Traces From Mechanical Insuflattion:Exsufflation (MI:E) be Used to Identify Laryngeal Responses to MI:E and Thus Optimise Treatment Algorithms?
This study looks at how a 'cough assist' machine works for people with muscle-weakening conditions. Researchers want to see if they can use the machine's readouts to understand how a patient's throat reacts and improve how therapists use the machine.
United Kingdom - recruitingNA
Optimisation of Mechanical Insufflation:Exsufflation
This study looks at how to make devices that help clear mucus from the lungs work better for people with muscle-weakening conditions. We want to find the best settings for these devices to help patients breathe easier and stay healthier.
United Kingdom - not yet recruitingNA
Nebulised Hypertonic Saline to Decrease Respiratory Exacerbations in Neuromuscular Disease or Neurodisability
This study looks at whether breathing in salty water (hypertonic saline) can help people with certain muscle or brain conditions avoid frequent chest infections. Participants will use a nebuliser twice a day for a year to see if this treatment reduces the need for antibiotics.
United Kingdom - active not recruitingPHASE3
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
This study looks at a new medicine called taldefgrobep alfa for young people with Spinal Muscular Atrophy (SMA). It's for those already on other SMA treatments to see if adding this new medicine can further improve muscle strength and movement.
United States · Belgium · Czechia - enrolling by invitation
MYO-SHARE: MYO-MRI in Neuromuscular Diseases
This study, MYO-SHARE, is looking at how MRI scans of muscles can help doctors diagnose neuromuscular diseases more accurately. It aims to create a better way to use these scans to understand different muscle conditions, making diagnosis easier and less invasive for patients.
Canada - active not recruitingPHASE3
Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab
This study looks at the long-term safety and effects of a medicine called apitegromab for people with Spinal Muscular Atrophy (SMA) types 2 and 3. It's for those who have already completed earlier studies with this drug.
United States · Belgium · France - recruiting
MSOT as Non-invasive Biomarker for Diagnosis and Monitoring of Neuromuscular Diseases
This study looks at a new, gentle scanning method called MSOT to diagnose and track muscle diseases in children. It aims to see if MSOT can measure important things in muscles without needles or surgery, helping doctors understand how these conditions develop.
Canada - recruiting
Research Biobank From Neuromuscular Biopsy Residues (in the Context of Care)
This study collects leftover muscle and nerve tissue from routine biopsies at Bordeaux University Hospital. It's for people with muscle or nerve conditions. The aim is to build a high-quality collection of samples to help researchers better understand these diseases and find new treatments. Your participation involves no extra procedures.
France - recruitingNA
Respiratory Muscles and Work of Breathing in Children
This study looks at how well children's breathing muscles work, especially if they have conditions affecting these muscles. We use a special tube to measure muscle strength and how hard they have to work to breathe. This helps doctors understand their breathing problems better and choose the best treatments.
France - not yet recruiting
Physical Activity Level at Home in CMT1A Patients: Wearable Sensor Assessment
This study looks at how useful small wearable sensors are for understanding daily activity in people with Charcot-Marie-Tooth disease type 1A (CMT1A). It aims to see if these sensors can measure movement as well as traditional clinic tests, potentially making it easier to keep track of the condition at home.
France - recruiting
Investigating Health Related Quality of Life in Patients With Chronic Respiratory Failure
This study looks at how chronic respiratory failure affects daily life for people in the UK. We want to understand their quality of life and how different treatments help.
United Kingdom - recruitingPHASE2
Assessment of Volume-targeted Ventilation in Patients With Neuromuscular Disease
This study looks at a new type of breathing support, called volume-targeted non-invasive ventilation (VT-NIV), for people with muscle conditions that affect their breathing. We want to see if it's safe and works well to help manage carbon dioxide levels, compared to the usual treatment.
United Kingdom