Quality of Life Outcomes According to Differential Nusinersen Exposure in Pediatric Spinal Muscular Atrophy

• Autori principali: Meaghann S. Weaver (Autore), Alice Yuroff (Autore), Sarah Sund (Autore), Scott Hetzel (Autore), Matthew A. Halanski (Autore)
• Natura: Libro
• Pubblicazione: MDPI AG, 2021-07-01T00:00:00Z.
• Soggetti: article
• Accesso online: Connect to this object online.

The purpose of this study was to explore early changes in patient and family caregiver report of quality of life and family impact during the transitional period of nusinersen use. Communication; family relationships; physical, emotional, social, and cognitive functioning; and daily activities were...