Successful weaning from mechanical ventilation in a patient with SMA type 1 treated with nusinersen | Zendy

Lee Jiwon | Zendy; Park Se Eun | Zendy; Lee Dajeong | Zendy; Song Joo Young | Zendy; Lee Jeehun | Zendy

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Successful weaning from mechanical ventilation in a patient with SMA type 1 treated with nusinersen

Author(s) -

Lee Jiwon,

Park Se Eun,

Lee Dajeong,

Song Joo Young,

Lee Jeehun

Publication year - 2021

Publication title -

annals of clinical and translational neurology

Language(s) - English

Resource type - Journals

SCImago Journal Rank - 1.824

H-Index - 42

ISSN - 2328-9503

DOI - 10.1002/acn3.51321

Subject(s) - medicine , sma* , weaning , mechanical ventilation , assisted ventilation , ventilation (architecture) , surgery , pediatrics , anesthesia , mechanical engineering , mathematics , combinatorics , engineering

SMA type 1 is the most severe type, characterized by early onset at <6 months of age, and rapid progression resulting in permanent assisted ventilation before 2 years of life. Supportive care was the only treatment until the approval of nusinersen, an antisense oligonucleotide drug that increases functional SMN protein levels. We present a case of successful weaning from permanent ventilation via tracheostomy with nusinersen in an infant who had been diagnosed with SMA type 1 at the age of one month and had become ventilator‐dependent from the age of 3 months.

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