Patient case (clinical description): An 8-year-old boy is brought to the clinic by his parents because of weakness and difficulty of standing up from a sitting position. The mother is healthy but had a brother who died in his 20th after being disabled and using wheelchairs in the last few years of his life. Physical examination shows 3/5 lower extremity muscle strength and enlarged calf muscles. The other physical examination and vital signs are unremarkable. Muscle biopsy showed absence of dystrophin protein. The patient is diagnosed with DMD.

Patient's own account: I brought my  8-year-old son to the doctor's clinic. He was struggling to stand up from a chair for the past few days. My wife is healthy but she was pretty worried since his brother died in his 20s after being disabled and using a wheelchair for the last years of his life. The doctor did an exam which highlighted weakness in the leg muscles and unusually large calf muscles. The rest of the exam was fine, and his vitals were normal. My son did a muscle biopsy, and it showed that the protein important for muscle strength and function is missing. The doctor diagnosed him with Duchenne muscular dystrophy.

Clinical trial: A Low Interventional Study to Monitor Activity Using Wearable Sensors in Duchenne Muscular Dystrophy

Eligibility criteria:
Inclusion Criteria:

          -  Diagnosis of Duchenne muscular dystrophy confirmed by medical history and genetic
             testing

          -  Body weight between 15 and 50 kg

          -  Receipt of glucocorticoids for 6 months and a stable daily dose for at least 3 months
             prior to study entry

          -  Ability to rise from floor within seven (7) seconds and ability to walk

        Exclusion Criteria:

          -  Current exposure to systemic immunosuppressant agents other than glucocorticoids.

          -  Prior exposure to any gene therapy agent, including exon-skipping and missense agents.

          -  Exposure to other investigational drugs within 30 days or 5 half-lives, whichever is
             longer.

          -  Any injury which may impact functional testing per investigator's judgement. Previous
             injuries must be fully healed prior to consenting. Prior lower limb fractures must be
             fully healed and at least 3 months from injury date at screening.

          -  Any planned surgeries which may impact physical activity and performance.

          -  Presence or history of musculoskeletal or neurological disease in addition to DMD.

          -  Any known allergies or skin reactions to stainless steel, versaflex, and silicon that
             may cause possible discomfort by wearable sensors.

          -  Evidence or history of clinically significant hematological, renal, endocrine,
             pulmonary, gastrointestinal, cardiovascular, hepatic, cancer, autoimmune or allergic
             disease that may interfere with the study conduct as per investigator's judgment,
             excluding untreated, asymptomatic, seasonal allergies at time of screening.

          -  Investigator site staff members directly involved in the conduct of the study and
             their family members, site staff members otherwise supervised by the investigator, or
             participants who are Pfizer employees, including their family members, directly
             involved in the conduct of the study.
Male
No healthy subjects accepted to join the trial.
Subject must be at least 4 Years old.
Subject must be at most 12 Years

Eligibility (NLI label): Entailment