Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen
study id #: NCT04602195
condition: Spinal Muscular Atrophy
status: Recruiting
purpose:The Motor Function Measure (MFM), a reliable tool assessing motor function and its progression in most neuromuscular diseases, is widely used in France in many teams. It can be used regardless of the severity of the motor impairment or the ambulatory status of the patient, allowing its use throughout the whole follow-up period of the patient, even in case of the loss of walking. Two versions of the MFM exist, one composed of 32 items originally validated for patients from 6 years old (MFM-32) and a shorter version composed of 20 items originally validated for patients between 2 and 6 years old (MFM-20).
In order to prove the possible use of MFM-32 as early as the age of 2 years to validly and reliably monitor the evolution of the motor function of children treated with Nusinersen, we propose in this project to study the sensitivity to treatment-induced change of MFM-32 and the validity of the scale in this population.
results: https://clinicaltrials.gov/ct2/show/results/NCT04602195
last updated: February 04, 2022
-
Sleep Breathing Disorders in Pediatric Patients With Spinal Muscular Atrophy 2Objectives: The study is aimed to analyz...
-
Spinal Muscular Atrophy Patient iPSC-Derived Motor Neurons Display Altered Proteomes at Early Stages of Differentiat...Spinal muscular atrophy (SMA) is an auto...
-
Kathrin C. Meyer, PhDKathrin C. Meyer, PhD, studied Cellular ...
-
Understanding Spinal Muscular Atrophy (SMA)https://www.youtube.com/watch?v=5mI_ZsWk...
-
Matthew Harms, MDMatthew Harms, MD is an Associate Profes...
-
Claire Altman Heine FoundationThe Claire Altman Heine Foundation (CAHF...
-
The SMA France National Registry: Already Encouraging ResultsSpinal muscular atrophy is a debilitatin...