Natural history of Type 2 and 3 spinal muscular atrophy: 2-year NatHis-SMA study.

ABSTRACT:

Objective

To characterize the natural history of spinal muscular atrophy (SMA) over 24 months using innovative measures such as wearable devices, and to provide evidence for the sensitivity of these measures to determine their suitability as endpoints in clinical trials.

Methods

Patients with Type 2 and 3 SMA (N = 81) with varied functional abilities (sitters, nonsitters, nonambulant, and ambulant) who were not receiving disease-modifying treatment were assessed over 24 months: motor function (Motor Function Measure [MFM]), upper limb strength (MyoGrip, MyoPinch), upper limb activity (ActiMyo^® ), quantitative magnetic resonance imaging (fat fraction [FF_T2 ] mapping and contractile cross-sectional area [C-CSA]), pulmonary function (forced vital capacity

SUBMITTER: Annoussamy M 

PROVIDER: S-EPMC7886049 | biostudies-literature | 2021 Feb

REPOSITORIES: biostudies-literature