64 results
Primary ObjectiveThe primary objective of this study is to examine the feasibility of using the clubfoot brace. This will be done by recording the effect of the brace on the clubfoot deformity by determining the improvement of the Dimeglio and…
To investigate the difference in muscle-tendon parameters of 2-11 years old unilateral clubfoot patients by means of 3D ultrasound. Subaims investigate the relation between muscle-tendon properties and age/ functional/clinical status of the clubfoot…
This study has been transitioned to CTIS with ID 2023-504520-26-00 check the CTIS register for the current data. Primary objective:To assess the long term safety and tolerability of GIVINOSTAT in patients with DMD following core protocols program…
Primary objective* To determine the extent to which muscle strength is affected in patients with RYR1-RM with autosomal dominantmutations and describe measurements over time.Secondary objectives* To determine the extent to which fatigue and physical…
Obtain longterm follow-up data in a large and clinically and genetically well-characterized cohort and thereby contributing to knowledge about the disease course and clinical trial readiness for FSHD research.
The primary objective is to determine if physical fatigability is present in infantile and young adult patients with FSHD.The secondary objectives are to determine if there is an association between fatigability ando Disease severityo Muscle…
This study has been transitioned to CTIS with ID 2023-509935-23-00 check the CTIS register for the current data. Part A:To evaluate the safety and tolerability of multiple ascending doses of SRP-5051 (4 mg/kg, 10 mg/kg, 20 mg/kg, and 30 mg/kg),…
Motorized arm support augmenting upper extremity function of people with Duchenne Muscular Dystrophy
The primary objectives of the different substudies are: 1) to identify the level and behavior of passive forces in the arm2) compare different weight and joint impedance compensation strategies of the motorized arm support3) evaluate the overall…
This study has been transitioned to CTIS with ID 2024-518686-10-00 check the CTIS register for the current data. To describe and investigate safety and tolerability of the intradermal delivery of two fractional doses of 3/3 µg Comirnaty Original/…
This study has been transitioned to CTIS with ID 2023-505170-15-00 check the CTIS register for the current data. The objective of the study is to assess the long-term safety and efficacy of intravenous ATB200 co-administration with oral AT2221 in…
Clinical, biochemical and radiological characterization of Van Buchem Disease
The goal of this study is to perform an additional follow-up visit on the FSHD-FOCUS cohort one year after the five year follow-up visit to assess the responsiveness over one and a half year, a reasonable timeframe for a clinical trial, of highly…
To objectively determine the severity of CTD related physical impairments, complaints and difficulties during participation in daily life activities. An additional study objective is: - To determine risk factors for the development of health…
This study has been transitioned to CTIS with ID 2024-512736-29-00 check the CTIS register for the current data. Primary Objective1. To evaluate the safety and tolerability of long-term dosing of losmapimod tablets in subjects with FSHD1Secondary…
Primary Objective: To study in detail whether a 12-week home based personalized IMT training scheme using the POWERbreathe KHP2 in DM1 patients could result in improvement of maximum inspiratory pressure (PImax) and endurance capacity. We postulate…
1. Compare molecular signatures in six leg muscles using RNA-sequencing;2. Correlate these signatures to the muscle involvement pattern in three muscular dystrophies and identify regulators for muscle strength preservation
Primary objective:• To evaluate the safety and tolerability of WVE-210201Secondary objectives:• To evaluate the effect of WVE-210201 on dystrophin production• To evaluate the concentration of WVE-210201 in plasma following treatment with WVE-210201…
Obtain longterm follow-up data in a large and clinically and genetically well-characterized cohort and thereby contributing to knowledge about the disease course and clinical trial preparedness for FSHD research.
The purpose of the study is to evaluate changes in key clinical outcome measures (eg, motor, respiratory, fatigue) in adult subjects with late-onset Pompe disease (LOPD) receiving standard-of-care enzyme replacement therapy (ERT). Additionally,…
To compare gait and balance measured by the traditional 3D gait analysis system (VICON) and by the new system including a treadmill and a virtual environment (CAREN) during self- paced walking in typically developed children. In addition, a…