"I have SMA, SMA doesn't have me": a qualitative snapshot into the challenges, successes, and quality of life of adolescents and young adults with SMA

Mazzella, A; Curry, M; Belter, L; Cruz, R; Jarecki, J

Mazzella, A (corresponding author), Cure SMA, 925 Busse Rd, Elk Grove Village, IL 60007 USA.

ORPHANET JOURNAL OF RARE DISEASES, 2021; 16 (1):

Abstract

Background With the approval of three treatments for spinal muscular atrophy (SMA) and several promising therapies on the horizon, the SMA adolescent ......

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