EP.82A prospective study of eteplirsen treatment for Duchenne muscular dystrophy in routine clinical practice: study design and methodology

2019 ◽  
Vol 29 ◽  
pp. S176
Author(s):  
K. Tsai ◽  
R. Fischer ◽  
B. Chao ◽  
Y. Zheng ◽  
K. Moitoso ◽  
...  
2019 ◽  
Vol 29 ◽  
pp. S173
Author(s):  
S. Geuens ◽  
M. Verly ◽  
J. Lemiere ◽  
N. Goemans ◽  
L. De Waele

Bone Reports ◽  
2020 ◽  
Vol 13 ◽  
pp. 100591
Author(s):  
Wenbin Zheng ◽  
Yi Dai ◽  
Jing Hu ◽  
Dichen Zhao ◽  
Ou Wang ◽  
...  

2019 ◽  
Vol 26 (14) ◽  
pp. 1898-1906 ◽  
Author(s):  
Laura Couloume ◽  
Laetitia Barbin ◽  
Emmanuelle Leray ◽  
Sandrine Wiertlewski ◽  
Emmanuelle Le Page ◽  
...  

Background: A recent controlled trial suggested that high-dose biotin supplementation reverses disability progression in patients with progressive multiple sclerosis. Objective: To analyze the impact of high-dose biotin in routine clinical practice on disability progression at 12 months. Methods: Progressive multiple sclerosis patients who started high-dose biotin at Nantes or Rennes Hospital between 3 June 2015 and 15 September 2017 were included in this prospective study. Disability outcome measures, patient-reported outcome measures, relapses, magnetic resonance imaging (MRI) data, and adverse events were collected at baseline, 6, and 12 months. Results: A total of 178 patients were included. At baseline, patients were 52.0 ± 9.4 years old, mean Expanded Disability Status Scale (EDSS) score was 6.1 ± 1.3, mean disease duration was 16.9 ± 9.5 years. At 12 months, 3.8% of the patients had an improved EDSS score. Regarding the other disability scales, scores either remained stable or increased significantly. In total, 47.4% of the patients described stability, 27.6% felt an improvement, and 25% described a worsening. Four patients (2.2%) had a relapse. Of the 74 patients (41.6%) who underwent an MRI, 20 (27.0%) had new T2 lesions, 8 (10.8%) had gadolinium-enhancing lesions. Twenty-five (14%) reported adverse event. Conclusion: In this study, high-dose biotin did not seem to be associated with a clear improvement in disability.


2017 ◽  
Vol 88 (5) ◽  
pp. e1.45-e1
Author(s):  
Steve Vucic ◽  
Michael H Barnett ◽  
Stefan Blum ◽  
Neil Shuey ◽  
Richard Worrell ◽  
...  

2021 ◽  
Vol 429 ◽  
pp. 119205
Author(s):  
Alfredo D’Aniello ◽  
Anna Teresa Giallonardo ◽  
Oriano Mecarelli ◽  
Umberto Aguglia ◽  
Giovanni Assenza ◽  
...  

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