First drug registry in Duchenne muscular dystrophy (DMD) to assess Translarna (Ataluren) use, safety, and effectiveness in routine clinical practice

2015 ◽  
Vol 25 ◽  
pp. S259
Author(s):  
K. Bushby ◽  
A. Reha ◽  
V. Northcutt ◽  
X. Luo ◽  
T. Ong ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Routine Clinical Practice ◽  
Drug Registry

EP.71Behavioral problems in Duchenne muscular dystrophy: how do we manage them in clinical practice?

2019 ◽  
Vol 29 ◽  
pp. S173
Author(s):  
S. Geuens ◽  
M. Verly ◽  
J. Lemiere ◽  
N. Goemans ◽  
L. De Waele
Keyword(s):  
Clinical Practice ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy

scholarly journals Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry

2019 ◽  
Vol 8 (14) ◽  
pp. 1187-1200 ◽  
Author(s):  
Francesco Muntoni ◽  
Isabelle Desguerre ◽  
Michela Guglieri ◽  
Andrés Nascimento Osorio ◽  
Janbernd Kirschner ◽  
...  
Keyword(s):  
Standard Deviation ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Real World ◽  
Nonsense Mutation ◽  
Duchenne Muscular Dystrophy Patient ◽  
Patient Demographics ◽  
Drug Registry ◽  
Real World Evidence ◽  
Multicenter Registry

Aim: Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (DMD) in clinical practice (NCT02369731). Here, we describe the initial demographic characteristics of the registry population. Patients & methods: Patients will be followed up from enrollment for ≥5 years or until study withdrawal. Results & conclusion: As of 9 July 2018, 213 DMD boys were enrolled from 11 countries. Mean (standard deviation) ages at first symptoms and at study treatment start were 2.7 (1.7) years and 9.8 (3.7) years, respectively. Corticosteroids were used by 190 patients (89.2%) before data cut-off. Mean (standard deviation) ataluren exposure was 639.0 (362.9) days. Six patients withdrew. STRIDE is the first drug registry for patients with DMD and represents the largest real-world registry of patients with nmDMD to date.

scholarly journals Clinical practice with steroid therapy for Duchenne muscular dystrophy: An expert survey in Asia and Oceania

2020 ◽  
Vol 42 (3) ◽  
pp. 277-288
Author(s):  
Fumi Takeuchi ◽  
Harumasa Nakamura ◽  
Naohiro Yonemoto ◽  
Hirofumi Komaki ◽  
Raymond L. Rosales ◽  
...  
Keyword(s):  
Clinical Practice ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Steroid Therapy ◽  
Expert Survey

Natural History of Serum Enzyme Levels in Duchenne Muscular Dystrophy and Implications for Clinical Practice

2020 ◽  
Vol 99 (12) ◽  
pp. 1121-1128
Author(s):  
Maricela Rodríguez-Cruz ◽  
Tomas Almeida-Becerril ◽  
Salvador Atilano-Miguel ◽  
Alan Cárdenas-Conejo ◽  
Mariela Bernabe-García
Keyword(s):  
Clinical Practice ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Natural History ◽  
Serum Enzyme ◽  
History Of
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