This trial is evaluating whether SRP-5051 will improve 3 primary outcomes and 14 secondary outcomes in patients with Muscular Dystrophies. Measurement will happen over the course of Part B predose and at multiple timepoints (up to 24 hours) after end of infusion.
This trial requires 60 total participants across 2 different treatment groups
This trial involves 2 different treatments. SRP-5051 is the primary treatment being studied. Participants will be divided into 2 treatment groups. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.
"Overall, our study shows some initial indication that patients with muscular dystrophies can have a longer survival time after the onset of the illness if treated early. Patients with muscular dystrophies can also reap some important benefits and improvements in their quality of life after having their muscles weakened by the illness, although the data are still limited." - Anonymous Online Contributor
"The main genetic abnormalities are deletions or duplications of a segment of the muscle protein collagen III (collagen III), or deletions causing a change in the location of the collagen III gene. These abnormalities are present in less than 50% of people with one of the three types of muscular dystrophy." - Anonymous Online Contributor
"While most children and adolescents with muscular dystrophy have mild clinical characteristics, in rare cases muscle weakness, hypotonia, and other non-neurological problems may be seen. Further skeletal imaging, echocardiography, electrocardiography (ECG), and cardiac echocardiography should be considered in these patients." - Anonymous Online Contributor
"The signs of limb-girdle muscular dystrophies include limb shortness, weakness, and atrophy. The signs of limb-girdle muscular dystrophies are best understood if viewed in terms of the functional impairment that is apparent in the affected limb(s). Many of the signs of muscular dystrophies were not only perceived, but were also assessed on a numerical rating scale." - Anonymous Online Contributor
"There are about 1 million Americans affected by one type of muscular dystrophy, Duchenne muscular dystrophy; and about 1 in 20,000 of US males is affected. These figures may be lower, given that a number of patients may have only minor symptoms. Muscle Diseases (Muscular Dystrophies, Infantile, Juvenile, Spinal Muscular Atrophy, X-Linked Hypophosphatemic Spondyloepiphyseal Dysplasia) account for 40% of all forms of muscular dystrophy in the United States and about 20% of all forms worldwide." - Anonymous Online Contributor
"Patients with muscle dystrophies should receive a multidisciplinary approach to symptom management and to coordinate care with their healthcare teams. Therapies for muscle dystrophies should include manual therapy techniques." - Anonymous Online Contributor
"ssp is a novel immunomodulator. In clinical trials, ssp was associated with a significant, early, yet clinically modest, improvement in muscle quality and strength in patients with multiple sclerosis." - Anonymous Online Contributor
"MD shows strong heritability, especially for MFCS. This supports the presence of modifying genes. It may be helpful in understanding the etiopathogenesis. We did not replicate previous data that the mutation carrier status for MD is higher in families." - Anonymous Online Contributor
"The most severe muscular dystrophies can be considered as the most promising ones to be treated with the most effective pharmacological therapy in order to modify the progression of the disease and to prevent the onset of muscle loss." - Anonymous Online Contributor
"Serum Response Factor - 5041 provided significant improvements in functional status and muscle strength over 12 weeks in all randomized muscle-skeletal patients in the double-blind placebo-controlled phase of SERENDIA trial when compared to conventional care and to placebo in an open label extension trial." - Anonymous Online Contributor
"Data from a recent study showed that Srp-5051 was frequently used in combination with other treatments. Future studies will need to assess the efficacy of a single treatment with Srp-5051 or multiple treatments with Srp-5051 in the treatment of LEMS." - Anonymous Online Contributor
"In this animal study, sarafutrast-PDT significantly enhanced the efficacy of both conventional and hyperstimulation-based treatments for muscles of dystrophic mdx mice. This suggests that sarafutrast-PDT may be useful to treat mdx patients with skeletal muscle damage." - Anonymous Online Contributor