Article
Natural history in spinal muscular atrophy Type I in Taiwanese population: A longitudinal study.
Brain & development - 1 Jan 2021
Ou Shan-Fu, Ho Che-Sheng, Lee Wang-Tso, Lin Kuang-Lin, Jones Cynthia C, Jong Yuh-Jyh
Abstract excerpt
INTRODUCTION: Spinal muscular atrophy (SMA) is caused by a defect in the survival motor neuron 1 (SMN1) gene. The Cooperative Study of the natural history of SMA Type I in Taiwan is a retrospective, longitudinal, observational study that helps in further understanding SMA disease progression in patients who have not received disease-modifying therapeutic interventions. METHODS: Case report forms were used to...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
