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

Abstract

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.

Safety issues and harmful pharmacological interactions of nutritional supplements in Duchenne muscular dystrophy: considerations for Standard of Care and emerging virus outbreaks

“…to prevent and control cardiovascular complication (e.g. ACE inhibitors, beta-blockers) or other pathology processes, or new drugs when patients take part in clinical trials [5][6][7]31]. Polytherapy should also take into account supplements, such as the above-mentioned vitamin D to delay steroid-induced osteoporosis.…”

“…DMD patients treated with drugs or other phenol-based nutraceuticals metabolized by UGT may, therefore, incur in unwanted interactions with GTE [128]. For instance, UGT1A9 is also the major enzyme that metabolizes ataluren, a novel drug recently approved for DMD to promote ribosomal read-through for patients with premature stop codon mutations [31,129]. Its metabolism could be delayed or impaired by GTE, with potential toxic effects.…”

Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

“…STRIDE is the first drug registry for patients with nmDMD that will generate data on patterns of ataluren use and long-term patient outcomes in real-world routine clinical practice. Previously, we described the initial demographic characteristics of the study population in the STRIDE Registry, as of 9 July 2018 [15]. The Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS; ClinicalTrials.gov identifier: NCT00468832) was a prospective, longitudinal study of more than 400 patients with DMD who were followed up between 2006 and 2016 at 20 worldwide centers as part of the academic clinical trial network, CINRG [16,17].…”

Treatment with Ataluren for Duchene Muscular Dystrophy

“…This phenomenon is probably related to the sequential genetic testing process for DMD introducing delays in diagnosis. However, compared with five years ago, next-generation sequencing is now more accessible and less expensive; thus, performing the second step in the genetic testing process is more feasible now, closing this diagnostic delay [3].…”

“…Overall, the results corroborate previous evidence that ataluren treatment can slow disease progression in nmDMD. The STRIDE Registry contains patients with a broader range of ages and ambulatory ability than those in clinical trials, and thus, the data represents a broader range of real-world experiences [3].…”