Early treatment of type II SMA slows rate of progression of scoliosis.

Giorgia CorattiJacopo LenkowiczMaria Carmela PeraAdele D'AmicoClaudio BrunoConsolato GullìNoemi BrolattiMarina PedemonteLaura AntonaciMartina RicciAnna CapassoGianpaolo CicalaCostanza CutronaRoberto de SanctisSara CarnicellaNicola ForcinaMichela CaterucciaMaria Beatrice DamasioLuca LabiancaFrancesca ManfroniAntonio LeoneEnrico BertiniMarika PaneStefano PatarnelloVincenzo ValentiniEugenio Mercuri

Published in: Journal of neurology, neurosurgery, and psychiatry (2023)

This study highlights that pharmacological treatment, if initiated early, may slow down the progression of scoliosis in type II SMA patients. Larger studies are warranted to further investigate the effectiveness of individual pharmacological treatment on scoliosis progression in this patient population.

Keyphrases

randomized controlled trial
newly diagnosed
ejection fraction
combination therapy
case report
patient reported