Dysphagia in Children with Spinal Muscular Atrophy

Dysphagia in Children with Spinal Muscular Atrophy - DySMA

Status

Active, not recruiting

Phases

Unknown

Study type

Observational

Source

DRKS

Registry ID

DRKS00029541

Enrollment

Registered

2022-07-04

Start date

2022-05-17

Completion date

Unknown

Last updated

2025-12-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Infantile spinal muscular atrophy, type I [Werdnig-Hoffman] DYSPHAGIA

Interventions

Group 1: Clinical swallowing examination at 5 measurement points from the start of therapy, in infants and young children with SMA (0 to 3 SMN2 copies), as part of the currently approved therapies. Th

Eligibility

Sex/Gender

All

Age

0 Days to 24 Months

Inclusion criteria

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

Exclusion criteria: Non-consent of the parents

Design outcomes

Primary

Measure	Time frame
Development of Swallowing: In infants and young children with spinal muscular atrophy for at least 18 months after starting gene-modulating or gene-replacement therapy	—

Secondary

Measure	Time frame
Dysphagia, Pneumonia, and severe adverse events	—

Countries

Germany

Contacts

Public ContactJana Zang

Universitätsklinikum Hamburg-EppendorfKlinik und Poliklinik für Hör-, Stimm- und Sprachheilkunde

jana.zang@uke.de040-7410-52361

Outcome results

None listed

Source: DRKS (via WHO ICTRP) · Data processed: Feb 4, 2026