Background This qualitative study examined how individuals with Spinal Muscular Atrophy
(SMA), their caregivers, and clinicians defined meaningful change, primarily in the Type II …

Background Reports on the clinical meaningfulness of outcome measures in spinal
muscular atrophy (SMA) are rare. In this two-part study, our aim was to explore patients' and …

The advent of clinical trials in spinal muscular atrophy (SMA) has highlighted the need to
define patterns of progression using functional scales. It has recently been suggested that …

Abstract Introduction The Spinal Muscular Atrophy Health Index (SMA‐HI) is a multifaceted,
disease‐specific, patient‐reported outcome to measure an SMA patient's perception of their …

Introduction The natural history and treatment of spinal muscular atrophy (SMA) is currently
being transformed by the development and availability of novel therapies, with significant …

Background Spinal muscular atrophy (SMA) has profound implications across a lifetime for
people with the condition and their families. Those affected need long-term multidisciplinary …

Introduction There is a need to optimize the current clinical outcome measures in spinal
muscular atrophy (SMA) incorporating patients' and caregivers' perspectives. The aim of this …

The positive outcome of different therapeutic approaches for spinal muscular atrophy (SMA)
in clinical trials and in clinical practice have highlighted the need to establish if functional …

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 …

Introduction: To improve care for patients with spinal muscular atrophy (SMA), we assessed
the physical and mental quality of life (QoL) in 62 adult patients with SMA. Methods: Physical …