The effects of nusinersen treatment on respiratory status of children with spinal muscular atrophy.

Evrim HepkayaAyşe Ayzıt Kılınç Sakallıİpek ÜlkersoyAzer Kılıç BaşkanHüseyin ArslanÖzge MeralTuğçe Damla DilekSerhat GülerSema SaltıkHaluk Çokuğraş

Published in: Pediatrics international : official journal of the Japan Pediatric Society (2022)

We posit that nusinersen may change the natural prognosis of SMA and improve care of children with SMA. Following up children with SMA for longer periods under nusinersen may be beneficial for understanding the effects of treatment. Results of our study need to be supported by future long-term studies to reach a consensus on nusinersen, considering the overall genetic and environmental status as well as the cost-effectiveness of the treatment.

Keyphrases

young adults
palliative care
risk assessment
genome wide
smoking cessation