TY - JOUR
T1 - Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy
T2 - patient demographics and characteristics from the STRIDE Registry
AU - Muntoni, Francesco
AU - Desguerre, Isabelle
AU - Guglieri, Michela
AU - Osorio, Andrés Nascimento
AU - Kirschner, Janbernd
AU - Tulinius, Már
AU - Buccella, Filippo
AU - Elfring, Gary
AU - Werner, Christian
AU - Schilling, Traci
AU - Trifillis, Panayiota
AU - Zhang, Olivia
AU - Delage, Abdallah
AU - Santos, Claudio L
AU - Mercuri, Eugenio
PY - 2019/10
Y1 - 2019/10
N2 - 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.
AB - 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.
U2 - 10.2217/cer-2019-0086
DO - 10.2217/cer-2019-0086
M3 - Article
C2 - 31414621
VL - 8
SP - 1187
EP - 1200
JO - Journal of Comparative Effectiveness Research
JF - Journal of Comparative Effectiveness Research
SN - 2042-6305
IS - 14
ER -