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Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy — PHENO SMART  

Spinal Muscular Atrophy Clinical Trial

Official title:
Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy

Clinical Trial Summary

With the advent of new treatments for ASI, new phenotypes are emerging. The investigators propose to describe these new phenotypes by prospectively following children with ASI of all types treated with TRS and aged under 16 for 2 years. The investigators also propose to evaluate potential assessment tools to determine whether they are relevant for monitoring this population, either routinely or for future clinical trials. The investigators also aim to collect the total costs associated with ASI in order to propose a first prospective medico-economic study in France.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT06321965
Study type Interventional
Source Hospices Civils de Lyon
Contact Laure LE GOFF, PI
Phone 04.72.12.95.04
Email laure.le-goff@chu-lyon.fr
Status Not yet recruiting
Phase N/A
Start date April 1, 2024
Completion date October 1, 2027
View Details
See also
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