MDA 2020 abstract: Pulmonary function in non-ambulatory patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) from the STRIDE Registry and CINRG Duchenne Natural History Study: A matched cohort analysis
Get a brief comparison of pulmonary function decline between two matched cohorts of patients with Duchenne muscular dystrophy (DMD) in our abstract presented at MDA 2020
The objectives and approach taken in a matched cohort analysis comparing pulmonary function decline in non-ambulatory nmDMD patients treated with a mutation-specific DMD therapy plus standard of care (SoC; corticosteroid or palliative therapies) and matched patients with DMD receiving SoC alone (Cooperative International Neuromuscular Research Group [CINRG]) Duchenne Natural History Study are presented in this abstract
▼ Ataluren is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions via the national reporting system. Adverse events should also be reported to PTC at firstname.lastname@example.org.
Some cookies are essential, others help us improve your experience by providing insights into how the website is used. Select one or more of the cookie types listed below, and then save your preferences. Refer to our Cookies Statement for more information.
Performance cookies, which help us measure the website’s performance and improve your experience. In using performance cookies we do not store any personal data, and only use the information collected through these cookies in aggregated and anonymised form.