Objective: We sought to describe outcomes for SMA patients treated with onasemnogene abeparvovec (OA) from the Cure SMA 2020 Community Update Survey. Background: Limited real-world […]
Objective: We sought to describe out-of-pocket expenditures and time costs for families/caregivers of patients with SMA1, as well as health care resource use. Here, we […]
Scoliosis may develop with Spinal Muscular Atrophy (SMA) as well as typically developing adolescents, among other populations. Most literature describes scoliosis in non-ambulatory SMA versus […]
BACKGROUND & OBJECTIVES: Early, pre-symptomatic treatment of spinal muscular atrophy (SMA) with disease-modifying therapy improves outcomes. In the United States (US), newborn screening (NBS) for […]
Valosin-containing protein (VCP) associated multisystem proteinopathy (MSP) is a rare inherited disorder that may result in multisystem involvement of varying phenotypes including inclusion body myopathy, […]
Background: Radicava® (edaravone injection) is a US FDA-approved treatment for amyotrophic lateral sclerosis (ALS) that slows the rate of physical functional decline. As intravenous (IV) […]