M. Richard , R. Barrois , I. Desguerre , E. Deladrière , V. Leloup-Germa , C. Barnerias , C. Gitiaux
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引用次数: 0
Abstract
Background
Spinal muscular atrophy (SMA) is a neuromuscular disorder characterized by the degeneration of the anterior horn cells of the spinal cord. Nusinersen for the treatment of SMA has been covered by public healthcare in France since May 2017.
Objective
Our aim was to investigate whether there is a correlation between clinical and compound motor action potential (CMAP) measurements in SMA patients treated with nusinersen after 3 years’ follow-up.
Method
Motor skills were evaluated regularly between M0 and M36 using the Motor Function Measure (MFM) score. CMAP measurements were collected regularly between M0 and M22.
Results
Data for 10 patients with SMA type 2 were collected and divided into two age groups (< 5 years and > 5 years). Motor function improved, but not significantly, regarding distal motor skills (D3) in both groups, and in axial and proximal motor function (D2) in the younger group. CMAP measurements improved in all patients. CMAP increased significantly for the median nerve, and this improvement correlated significantly with global MFM and with axial and proximal tone (D2).
Conclusion
Our study shows gain in distal motor function with nusinersen, especially in younger patients with SMA type 2. These results encourage the screening of SMA patients and treatment as early as possible. CMAP measurements of the median nerve show clear improvement in patients treated with nusinersen and could be performed as routine follow-up.
期刊介绍：
Archives de Pédiatrie publishes in English original Research papers, Review articles, Short communications, Practice guidelines, Editorials and Letters in all fields relevant to pediatrics.
Eight issues of Archives de Pédiatrie are released annually, as well as supplementary and special editions to complete these regular issues.
All manuscripts submitted to the journal are subjected to peer review by international experts, and must:
Be written in excellent English, clear and easy to understand, precise and concise;
Bring new, interesting, valid information - and improve clinical care or guide future research;
Be solely the work of the author(s) stated;
Not have been previously published elsewhere and not be under consideration by another journal;
Be in accordance with the journal''s Guide for Authors'' instructions: manuscripts that fail to comply with these rules may be returned to the authors without being reviewed.
Under no circumstances does the journal guarantee publication before the editorial board makes its final decision.
Archives de Pédiatrie is the official publication of the French Society of Pediatrics.
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