Quantitative Assessment of Dysphagia in Spinal Muscle Atrophy (SMA) - DYS-SMA Trial

Status: Active, not recruiting
Location: See location...
Intervention Type: Diagnostic Test
Study Type: Interventional
Study Phase: Not Applicable

The major aim of this project is to assess comprehensively frequency and extent of dysphagia and bulbar dysfunction in SMA1, 2, and 3 patients by applying FEES and validated dysphagia scores. Further aims are to follow changes of dysphagia over time in newly diagnosed patients, and in subjects starting treatment with one of the new therapeutic SMA drugs. Special attention will be paid to subjects treated with Risdiplam. If applicable, the data will be compared between groups receiving different drugs.

Participation Requirements
Sex: All
Healthy Volunteers: No

⁃ All patients with genetically determined 5q-linked SMA followed at the neuromuscular center Gießen

• Informed and written consent signed by the patient or a legal guardian

Other Locations
University Hospital Giessen and Marburg, Campus Giessen
Time Frame
Start Date: June 1, 2020
Estimated Completion Date: June 1, 2023
Target number of participants: 79
Experimental: Spinal Muscle Atrophy
All patients with Spinal Muscle Atrophy type 1 to 4
Samra Hamzic
Leads: University of Giessen

This content was sourced from clinicaltrials.gov

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