Effect on maximal mouth opening in children with spinal muscular atrophy treated with onasemnogene abeparvovec

Spinal muscular atrophy (SMA) is a progressive, autosomal recessive neuromuscular disorder caused by a mutation of the SMN1 gene. This causes progressive muscle weakness and muscle atrophy. The voluntary muscles are affected, including those involved in swallowing and breathing. The muscles of mastication are also affected in them. Adequate mouth opening is required to maintain good oral hygiene and adequate mastication. Tools like maximum tongue pressure and maximum mouth opening (MMO) are often used as clinical and sensitive outcome measures for assessing oral function tests.1 MMO is the maximal interincisal distance on unassisted active mouth opening, reflecting the mandibular range of motion.2 Onasemnogene abeparvovec is the US Food and Drug Administration (FDA)-approved gene replacement therapy for SMA. There is little or no data on its effect on MMO or oral functions in the affected individuals, although positive changes are expected, as seen in the other...
Source: Archives of Disease in Childhood - Category: Pediatrics Authors: Tags: PostScript Source Type: research