MedPath

Dysphagia in Children with Spinal Muscular Atrophy

Recruiting
Conditions
DYSPHAGIE
G12.0
R13.9
Infantile spinal muscular atrophy, type I [Werdnig-Hoffman]
Registration Number
DRKS00029541
Lead Sponsor
niversitätsklinikum Hamburg-Eppendorf
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
Recruiting
Sex
All
Target Recruitment
60
Inclusion Criteria

Infants, toddlers, and children with 5-q associated spinal muscular atrophy with 0-3 SMN2 copies who are starting gene-modifying or gene-replacement therapy in Germany.

Exclusion Criteria

Non-consent of the parents

Study & Design

Study Type
observational
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Secondary Outcome Measures
NameTimeMethod
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