Eligibility: Patients aged 2 to 17 years old. Enrollment: Start of inclusion: Purpose: Open-label aiming at studying the safety, tolerability, and dose selection for a future trial of HT in boys with DMD. Principal Investigator or sponsor: Hoffmann-La Roche.
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What is spinal muscular atrophy? What causes SMA? How is it inherited? What are the types of SMA? How is SMA diagnosed? Are there treatments for SMA? What is the prognosis? What research is being done? Where can I get more information?
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View research View latest news Sign up for updates. Besides the direct effects of the muscular dystrophy, the increasing effort to perform activities, the fear of falling and the use of personal aids indirectly impair leg and arm functions as a result of disuse. Physical training could oppose this secondary physical deterioration. The No Use is Disuse NUD study is the first study in human subjects with DMD that will examine whether a low-intensity physical training is beneficial in terms of preservation of muscle endurance and functional abilities. The study consists of two training intervention studies: study 1 "Dynamic leg and arm training for ambulant and recently wheelchair-dependent boys with DMD and, study 2 "Functional training with arm support for boys with DMD who have been confined to a wheelchair for several years". This paper describes the hypotheses and methods of the NUD study. Study 1 is an explorative randomized controlled trial with multiple baseline measurements. The primary study outcomes are muscle endurance and functional abilities, assessed with a Six-Minute Bicycle Test and the Motor Function Measure. Study 2 has a within-group repeated measurements design and will include ten boys with DMD who have already been confined to a wheelchair for several years.
Spinal muscular atrophy is a genetic disorder characterized by weakness and wasting atrophy in muscles used for movement skeletal muscles. It is caused by a loss of specialized nerve cells, called motor neurons that control muscle movement. The weakness tends to be more severe in the muscles that are close to the center of the body proximal compared to muscles away from the body's center distal. The muscle weakness usually worsens with age. There are many types of spinal muscular atrophy that are caused by changes in the same genes. The types differ in age of onset and severity of muscle weakness; however, there is overlap between the types. Other forms of spinal muscular atrophy and related motor neuron diseases, such as spinal muscular atrophy with progressive myoclonic epilepsy , spinal muscular atrophy with lower extremity predominance , X-linked infantile spinal muscular atrophy , and spinal muscular atrophy with respiratory distress type 1 are caused by mutations in other genes. Spinal muscular atrophy type 0 is evident before birth and is the rarest and most severe form of the condition. Affected infants move less in the womb, and as a result they are often born with joint deformities contractures.