Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry
Por:
Muntoni F, Desguerre I, Guglieri M, Nascimento-Osorio A, Kirschner J, Tulinius M, Buccella F, Elfring G, Werner C, Schilling T, Trifillis P, Zhang O, Delage A, Santos CL and Mercuri E
Publicada:
1 oct 2019
Ahead of Print:
15 ago 2019
Categoría:
Health policy
Resumen:
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.
Filiaciones:
Muntoni F:
Dubowitz Neuromuscular Centre MRC Centre for Neuromuscular Diseases, University College London, Institute of Child Health Great Ormond Street Hospital for Children Foundation Trust, 30 Guildford Street, London WC1N 1EH, UK
NIHR Great Ormond Street Hospital Biomedical Research Centre, Great Ormond Street Institute of Child Health, University College London, Great Ormond Street Hospital Trust, London, UK
Desguerre I:
APHP Necker - Enfants Malades Hospital, Paris V Descartes University, Neuromuscular Network FILNEMUS, Paris, France
Guglieri M:
The John Walton Muscular Dystrophy Research Centre, Newcastle University Newcastle Upon Tyne Hospitals, Newcastle Upon Tyne, UK
Nascimento-Osorio A:
Hospital Sant Joan de Déu Unidad de Patología Neuromuscular, Universidad de Barcelona, CIBERER, ISCIII, Barcelona, Spain
Kirschner J:
Medical Center - University of Freiburg, Faculty of Medicine, Freiburg, Germany
Tulinius M:
Department of Pediatrics, Gothenburg University, Queen Silvia Children's Hospital, Gothenburg, Sweden
Buccella F:
Parent Project Italy APS, Rome, Italy
Elfring G:
PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA
Werner C:
PTC Therapeutics Germany GmbH, Frankfurt, Germany
Schilling T:
PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA
Trifillis P:
PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA
Zhang O:
PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA
Delage A:
PTC Therapeutics Switzerland GmbH, Zug, Switzerland
Santos CL:
PTC Therapeutics Inc., South Plainfield, NJ 07080-2449, USA
Mercuri E:
Department of Pediatric Neurology, Catholic University, Rome, Italy
Centro Clinico Nemo, Policlinico Universitario A Gemelli IRCCS, Rome, Italy
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