Publications by authors named "M L Main"
Article Synopsis
- - This study investigates the effects of scoliosis surgery on various outcomes (respiratory function, motor function, weight, pain, and patient satisfaction) in patients with spinal muscular atrophy (SMA), highlighting the lack of research on these secondary outcomes.
- - Results showed significant improvement in respiratory function for SMA2 patients postsurgery, but some experienced drops in gross motor scores and weight, with many reporting postsurgical pain, especially hip-related.
- - Despite these challenges, patients and parents expressed overall satisfaction with the surgery's impact on posture and appearance, emphasizing the need for better postoperative care, including physiotherapy and support services.
Article Synopsis
- Spinal muscular atrophy (SMA) is a genetic disorder leading to muscle weakness due to mutations in the SMN1 gene.* -
- Onasemnogene abeparvovec (OA) is a gene therapy approved for SMA that was administered to two heavy patients (20 kg) previously treated with another medication, Nusinersen.* -
- The patients experienced mixed results after receiving OA, including liver issues needing steroids and limited improvement in muscle function, highlighting the importance of weighing risks and benefits for older and heavier SMA patients.*
Background And Objectives: Nusinersen has shown significant functional motor benefit in the milder types of spinal muscular atrophy (SMA). Less is known on the respiratory outcomes in patients with nusinersen-treated SMA. The aim of this study was to describe changes in respiratory function in pediatric patients with SMA type 2 and 3 on regular treatment with nusinersen within the iSMAc international cohort and to compare their trajectory with the natural history (NH) data published by the consortium in 2020.
View Article and Find Full Text PDFBackground: Spinal muscular atrophy (SMA) is a neuromuscular disorder characterised by progressive motor function decline. Motor function is assessed using several functional outcome measures including the Revised Hammersmith Scale (RHS).
Objective: In this study, we present longitudinal trajectories for the RHS in an international cohort of 149 untreated paediatric SMA 2 and 3 patients (across 531 assessments collected between March 2015 and July 2019).