☆ 4.6 Article

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

JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY (2023)

相关参考文献

注意：仅列出部分参考文献，下载原文获取全部文献信息。

Article Medicine, Research & Experimental

Adherence and Persistence to Nusinersen for Spinal Muscular Atrophy: A US Claims-Based Analysis

David Fox et al.

Summary: This study aims to characterize real-world adherence and persistence to nusinersen treatment in patients with spinal muscular atrophy (SMA). The findings suggest that adherence and persistence to nusinersen treatment appear to be low, possibly influenced by patients' age and presence of spinal complications. Future research should explore possible reasons for low adherence and persistence.

ADVANCES IN THERAPY (2023)

添加到收藏夹

Article

Rehabilitation in Spinal Muscular Atrophy; A Narrative Review

Seth Kwame Agyenkwa et al.

European Journal of Therapeutics (2023)

添加到收藏夹

Article Respiratory System

Effect of nusinersen on respiratory function in paediatric spinal muscular atrophy types 1-3

Archana Chacko et al.

Summary: The first year of nusinersen treatment showed a reduced decline in lung function, especially in type 2 SMA patients, and an improvement in Apnoea-Hypopnoea Index (AHI).

THORAX (2022)

添加到收藏夹

Article Clinical Neurology

Scoliosis in Spinal Muscular Atrophy Type 1 in the Nusinersen Era

Fatima Al Amrani et al.

Summary: This study aims to investigate the development of scoliosis in patients with SMA1 treated with nusinersen. The study found that despite significant improvement in functional motor assessment, patients still experienced progression of scoliosis.

NEUROLOGY-CLINICAL PRACTICE (2022)

添加到收藏夹

Article Clinical Neurology

Long term follow-up of scoliosis progression in type II SMA patients

Giorgia Coratti et al.

Summary: The aim of this study is to retrospectively assess the onset and progression of scoliosis in untreated type II spinal muscular atrophy (SMA) patients. The results showed a positive correlation between age and scoliosis angles, with scoliosis progressively increasing with age. There was also a progressive increase in scoliosis angles with decreasing functional status scores. The longitudinal analysis revealed that age, functional status, and baseline scoliosis angle were predictive of scoliosis progression. The mean annual rate of increase in scoliosis angle was 5.63.

NEUROMUSCULAR DISORDERS (2022)

添加到收藏夹

Review Clinical Neurology

Mid- and long-term (at least 12 months) follow-up of patients with spinal muscular atrophy (SMA) treated with nusinersen, onasemnogene abeparvovec, risdiplam or combination therapies: A systematic review of real-world study data

Judit Erdos et al.

Summary: This systematic review aimed to evaluate the mid-and long-term effectiveness of approved drugs or combination therapies in different types of spinal muscular atrophy (SMA) patients. The results showed that Nusinersen, onasemnogene abeparvovec, and combination therapies improved motor function in SMA type 1 patients, while SMA type 2 to type 4 patients treated with Nusinersen showed stability or slight improvement. However, there were limited reports on quality of life measures, and the mid-and long-term effectiveness of Risdiplam could not be determined.

EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY (2022)

添加到收藏夹

Review Genetics & Heredity

Motor function in type 2 and 3 SMA patients treated with Nusinersen: a critical review and meta-analysis

Giorgia Coratti et al.

Summary: This review critically examined real-world data on motor function in SMA patients treated with Nusinersen, finding positive changes in the treated group compared to negative changes in the untreated group.

ORPHANET JOURNAL OF RARE DISEASES (2021)

添加到收藏夹

Review Orthopedics

Risdiplam for the Use of Spinal Muscular Atrophy

Juyeon Kakazu et al.

Summary: Currently, Risdiplam is the only orally administered drug approved by the FDA for the treatment of SMA, providing stronger systemic therapies and allowing for a non-invasive approach in infants. Further investigation and comparison on the safety profile of Risdiplam due to its broader systemic effect should be considered with other available therapies.

ORTHOPEDIC REVIEWS (2021)

添加到收藏夹

Article Pediatrics

Respiratory Needs in Patients with Type 1 Spinal Muscular Atrophy Treated with Nusinersen

Valeria A. Sansone et al.

JOURNAL OF PEDIATRICS (2020)

添加到收藏夹

Review Medicine, General & Internal

New Treatments in Spinal Muscular Atrophy: Positive Results and New Challenges

Sonia Messina et al.

JOURNAL OF CLINICAL MEDICINE (2020)

添加到收藏夹

Article Genetics & Heredity

Effects of nusinersen after one year of treatment in 123 children with SMA type 1 or 2: a French real-life observational study

Frederique Audic et al.

ORPHANET JOURNAL OF RARE DISEASES (2020)

添加到收藏夹

Article Pediatrics

Functional and surgical treatments in patients with spinal muscular atrophy (SMA)

C. Boulay et al.

ARCHIVES DE PEDIATRIE (2020)

添加到收藏夹

Article Orthopedics

VEPTR: Are We Reducing Respiratory Assistance Requirements?

Sarah B. Nossov et al.

JOURNAL OF PEDIATRIC ORTHOPAEDICS (2019)

添加到收藏夹

Article Clinical Neurology

Nusinersen in later-onset spinalmuscular atrophy Long-term results from the phase 1/2 studies

Basil T. Darras et al.

NEUROLOGY (2019)

添加到收藏夹

Article Clinical Neurology

Diagnosis and management of spinal muscular atrophy: Part 1: Recommendations for diagnosis, rehabilitation, orthopedic and nutritional care

Eugenio Mercuri et al.

NEUROMUSCULAR DISORDERS (2018)

添加到收藏夹

Article Orthopedics

Radiographic and Respiratory Effects of Growing Rods in Children With Spinal Muscular Atrophy

Rachel L. Lenhart et al.

JOURNAL OF PEDIATRIC ORTHOPAEDICS (2017)

添加到收藏夹

Article Clinical Neurology

Spinal Muscular Atrophy

Stephen J. Kolb et al.

NEUROLOGIC CLINICS (2015)

添加到收藏夹

Article Orthopedics

Early Treatment of Scoliosis With Growing Rods in Children With Severe Spinal Muscular Atrophy: A Preliminary Report

Sheila Chandran et al.

JOURNAL OF PEDIATRIC ORTHOPAEDICS (2011)

添加到收藏夹

Article Clinical Neurology

Growing Rods for Scoliosis in Spinal Muscular Atrophy Structural Effects, Complications, and Hospital Stays

Mark J. McElroy et al.

SPINE (2011)

添加到收藏夹

Article Clinical Neurology

Pelvic Fixation of Growing Rods Comparison of Constructs

Paul D. Sponseller et al.

SPINE (2009)

添加到收藏夹

Article Clinical Neurology

Consensus statement for Standard of Care in Spinal Muscular Atrophy

Ching H. Wang et al.

JOURNAL OF CHILD NEUROLOGY (2007)

添加到收藏夹

Article Clinical Neurology

The Hammersmith functional motor scale for children with spinal muscular atrophy: a scale to test ability and monitor progress in children with limited ambulation

M Main et al.

EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY (2003)

添加到收藏夹

© Peeref 2019-2024. All rights reserved.