Spinal muscular atrophy (SMA) patients. Spinal muscular atrophy and related syndromes
Conditions
Interventions
Intervention group: After reviewing the history of heart disease, patients will be given oral salbutamol for 12 weeks. The dose of the drug will be 0.3 to 0.4 mg/kg/day divided into two doses per day
Sponsors
Tehran University of Medical Sciences
Eligibility
Sex/Gender
All
Age
6 Months to 20 Years
Inclusion criteria
Inclusion criteria: Willingness to cooperate and sign the informed consent form after full knowledge of the objectives and methods of the study. Girls and boys aged 6 months to 20 years. Definitive genetic diagnosis of SMA 5q (homozygous gene mutation or deletion). Having two or three copies of SMN2.
Exclusion criteria
Exclusion criteria: Proven heart disease and blood pressure. The occurrence of any accident that affects a person's health. Acceptance rate less than 80%. Immigration Exclusion based on personal preference of participants or their parents Changes in medications taken during the study period
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Study of motor ability in patients. Timepoint: Beginning and end of the study. Method of measurement: According to the criteria ofHammersmith Functional Motor Scale Expanded (HFMSE).;Side effects of salbutamol. Timepoint: End of the study. Method of measurement: Clinical assessments and examinations. | — |
Secondary
| Measure | Time frame |
|---|---|
| Serum C-reactive protein (CRP) levels. Timepoint: Beginning and end of the study. Method of measurement: CRP ELISA Kit.;Body mass index. Timepoint: Beginning and end of the study. Method of measurement: Calculation using the ratio of weight (in kilograms) to the square of height (in centimeters). | — |
Countries
Iran (Islamic Republic of)
Contacts
Public ContactSara Memarian
Tehran University of Medical Sciences
Outcome results
None listed