A head-to-head study comparing the functional value of two models of robotically assisted rehabilitation in patients with SMA. A single-center, randomized, single-blinded, comparative study of Robotically Assisted Verticalization versus Robotically Assisted Locomotion.

The objective of research:

The main goal of the project is to determine the optimal robotically assisted rehabilitation model for people with SMA depending on age and baseline functional status. The study consists of a head-to-head comparison of two rehabilitation models.

1. Research period: 4 years
2. Patients age: 0-21 y.o.
3. Group size: 200 patients (100 patients in each group)
4. Assignment of patients to study groups in a randomised manner

Inclusion Criteria:

• Signing of informed consent to participate in the experiment by the participant or the participant's parent/legal guardian
• Diagnosis of SMA (spinal muscular atrophy 1-4) confirmed by genetic testing, symptomatic or pre-symptomatic
• Age between 0 and 21 years of age (will be determined on the day of starting participation in the project based on the date of birth)
• Treatment under a drug program for spinal muscular atrophy, provided that the patient has a diagnosis of spinal muscular atrophy at the date of eligibility.

Exclusion Criteria:

• Cardio-respiratory disorders requiring invasive ventilation
• Advanced osteoporosis with multiple fractures prior to treatment
• Functional deterioration during the rehabilitation process in the range of scales appropriately selected for the SMA type: Prechtl, HINE, CHOP-INTEND, HFMS, RULM
• Lack of cooperation with the therapist
• Other functional indications preventing exercise

Patients and their parents will not be blinded by the nature of the therapeutic procedure used after random allocation to the treatment group. Study personnel will be divided into Blinded Team and Unblinded Team to ensure objectivity of the assessment of safety and effectiveness of both strategies.