The aim of this study was to evaluate real-world treatment patterns and clinical outcomes in pediatric SMA patients in the Czech and Slovak Republics, using data from the Registry of muscular Dystrophy (REaDY).
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Number of Patients by Demographic Category
Timeframe: Baseline
Age at Diagnosis
Timeframe: Baseline
Age at Symptom Onset
Timeframe: Baseline
Weight
Timeframe: Baseline
Number of Patients by Medical Examination Result
Timeframe: Baseline
Blood Pressure
Timeframe: Baseline
Pulse
Timeframe: Baseline
Respiratory Rate
Timeframe: Baseline
Number of Patients by Motor Function
Timeframe: Baseline
Number of Patients by Feeding and Pulmonary Function
Timeframe: Baseline
FVC
Timeframe: Baseline
Number of Patients With a Physiotherapeutic Evaluation
Timeframe: Baseline
Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-Intend) Score
Timeframe: Baseline
Hammersmith Functional Motor Scale Expanded (HFMSE) Score
Timeframe: Baseline
Revised Upper Limb Module (RULM) Score
Timeframe: Baseline
6 Minute Walk Distance (6MWD)
Timeframe: Baseline
Number of Patients by Clinician's Rating of Patient's Current Severity of Illness
Timeframe: Baseline
Number of Patients by Change in Condition in the Last 6 Months as Assessed by the Clinician
Timeframe: Baseline
Number of Patients by Total Global Impression According to Patient or Parent
Timeframe: Baseline