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effectiveness and side effects of Spinaraza and Risidiplam on the motor performance of patients with spinal muscular atrophy

Not Applicable
Recruiting
Conditions
Spinal Muscular Atrophy.
Spinal muscular atrophy and related syndromes
Registration Number
IRCT20120821010634N4
Lead Sponsor
Hamedan University of Medical Sciences
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Recruiting
Sex
All
Target Recruitment
30
Inclusion Criteria

SMA clinical diagnosis by pediatric neurologist
SMA diagnosis according to genetic test

Exclusion Criteria

The patient do not have a consent

Study & Design

Study Type
interventional
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Assessment of Motor Function. Timepoint: one month, 3 months, and 6 months after drug initiation. Method of measurement: Assessment of motor function based on Hammersmith and HINE scales.;Clinical and laboratory side effects of drugs. Timepoint: one month, 3 months, and 6 month after drug initiation. Method of measurement: fulfilling check list according to patient reports, requesting CBC, liver and renal function tests.
Secondary Outcome Measures
NameTimeMethod
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