Selina S Vickery, BS; Jerry R Mendell, MD, Kelly Lehman, APN, Kan N. Hor, MD Background: Limb-Girdle Muscular Dystrophies (LGMD) are an inheritable group of […]
Background: Delandistrogene moxeparvovec (SRP-9001) is an investigational gene transfer therapy developed for targeted skeletal and cardiac muscle expression of micro-dystrophin—a shortened, functional dystrophin protein. In […]
Authors: Taylor Schwab, RN, Kaitlin Haug, RN, Christine Caneva, BS, Jennifer Coffman, DNP, CPNP-AP, Stefanie Leonard, MSN, RN, Melissa Gibbons, MS, CGC, Scott Demarest, MD, […]
Background: Myotonic dystrophy (DM) is the most common muscular dystrophy in adults. Dominantly inherited CTG and CCTG repeat expansions in DMPK and CNBP genes cause […]
Objective: We sought to describe real-world outcomes in US patients with SMA aged ?6 months treated with nusinersen monotherapy, onasemnogene abeparvovec (OA) monotherapy, or nusinersen […]
Objective: We sought to describe health care resource utilization (HCRU) for patients with spinal muscular atrophy (SMA) in the United States. Background: Limited real-world evidence […]
Objective: We sought to investigate the most important treatment characteristic preferences of US families and US health care professionals (HCPs) in choosing disease-modifying treatments (DMTs) […]
Objectives: We sought to describe health care resource utilization (HCRU), including hospitalizations, health care professional (HCP) visits, and device utilization, for children with SMA type […]
Objective: We sought to describe out-of-pocket expenditures and time costs as well as health care resource utilization (HCRU) for the families/caregivers of patients with SMA1. […]