Acetaminophen treatment in children and adults with spinal muscular atrophy: a lower tolerance and higher risk of hepatotoxicity

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disorder caused by mutations in the survival motor neuron 1 (SMN1) gene on chromosome 5q12 [1]. Bi-allele deficiency of SMN1 causes degeneration of the anterior horn cells of the spinal cord, resulting in progressive muscular weakness and atrophy. Based on the age of onset and disease severity, SMA is divided into subtypes from I to IV [1]. The natural history of untreated SMA type I and type II (SMA II) renders patients wheelchair dependent and unable to walk at any time [1].

Source: Neuromuscular Disorders - November 15, 2023 Category: Neurology Authors: Marie Mostue Naume, Qiaolin Zhao, Sissel Sundell Haslund-Krog, Thomas Krag, Brenda C.M.de Winter, Karoline Lolk Revsbech, John Vissing, Helle Holst, Morten Hylander M øller, Tessa Munkeboe Hornsyld, Morten Dunø, Christina Engel Høi-Hansen, Alfred Peter Tags: Research paper Source Type: research