Active clinical trials for "Muscular Dystrophies"

Patients at risk of developing respiratory dysfunctions, such as patients with severe forms of muscular dystrophy, need a careful respiratory assessment, and periodic follow-up visits to monitor the progression of the disease. Continuous monitoring of respiratory activity pattern at home could give additional understandings about disease progression, flanking traditional, intermittent, cardiopulmonary evaluations, allowing prompt clinical intervention, and anticipating respiratory dysfunction. The main objective of the present study is thus to investigate the feasibility of using an innovative wearable device for respiratory monitoring, especially breathing frequency variation assessment, in patients with muscular dystrophy. The comparison between the measurements of breathing frequency obtained by using the IMU-based device and by using the reference method provided optimal results, in terms of accuracy errors, correlation and agreement. Participants positively evaluated the device for what concerns ease of use, comfort, usability and wearability. Moreover, preliminary results confirmed that breathing frequency is an interesting breathing parameter to monitor, at the clinic and at home, because it strongly correlates with the main indexes of respiratory function

This study is intended to build on a growing body of literature showing a blood flow abnormality in patients with Becker muscular dystrophy. The investigators' laboratory recently showed that this blood flow abnormality could be corrected by a single oral dose of the drug Tadalafil (also known as Cialis). The investigators now wish to replicate these exciting results using a common nitric oxide donor (sodium nitrate).

People with mobility disabilities are at greater risk than the general population for incurring health problems. Many of these conditions are preventable through behavior and lifestyle changes such as exercise and physical activity. Recent evidence suggests that people with disabilities experience the same physiologic response to exercise as the general population. Nonetheless, nearly three-fourths of those with disabilities report being entirely sedentary or not active enough to achieve health benefits. Despite some knowledge of issues that limit physical activity among this population, few studies have investigated methods for promoting physical activity adoption among people with disabilities, including wheelchair users. The purpose of this study is to test the effectiveness of a behavioral intervention to promote physical activity adoption over 6 months and maintenance of physical activity over another 6 months by community-dwelling manual wheelchair users.

The study is a feasibility study or pilot study, that is a clinical investigation to acquire the preliminary information on a motorized exoskeleton (BRIDGE / EMPATIA exoskeleton) for the movement of the upper limb in order to develop it, including design changes. The primary objective of the clinical trial is to assess the fit, safety and usability of the device in supporting the execution of daily activities for patients suffering from muscular dystrophy. The risk analysis for the BRIDGE / EMPATIA device does not present particular criticalities that preclude the use of the device in the target population. In any case, during the trial eventual adverse events are recorded for the verification of safety..

The proposed clinical trial is an outgrowth of the safety record and functional improvement seen in the BMD follistatin gene therapy trial. In this study the investigators propose to inject AAV1.CMV.huFS344 at a total dose of 2.4E12 vg/kg to six DMD patients. This dose will be divided between gluteal muscles, quadriceps and tibialis anterior. This is a wider distribution of vector than given to BMD patients, who overall improved the distance walked on the 6MWT without adverse events related to viral transduction into a single muscle.

This study will collect MRI from healthy volunteer boys and boys with Duchenne Muscular Dystrophy (DMD) to help researchers identify and validate cardiac MRI biomarkers to better understand the health of the heart and changes in heart health over time in boys with DMD. Currently, there is a lack of sufficiently well characterized cardiac MRI biomarkers that can serve as endpoints for detecting on-target and/or off-target cardiac effects during clinical drug trials for boys with DMD. Consequently, the first objective is to identify and characterize several cardiac MRI biomarkers for boys with DMD.

Objective of this study is to evaluate safety and tolerability of flavocoxid administered at the daily oral dose of 500 or 1000 mg/die for one year in DMD patients, alone or in association with steroids (deflazacort on alternate days) started at least one year before. The investigators will also perform a multidimensional clinical evaluation covering functional and muscle strength and quality of life (QoL)assessments.

Study to characterize the single-state and steady-state dosing of oral deflazacort in pediatric and adolescents subjects.

Investigators investigated that the effects of kinesilogy taping on performance, energy consumption and gait characteristics in patients with Duchenne Muscular Dystrophy

The investigators aim to investigate the effect of high-intensity training in patients with facioscapulohumeral muscular dystrophy. Can patients benefit from this type of exercise without muscle damage.