CNS 2019 poster: Phase 2 trials to assess production of dystrophin protein in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) receiving ataluren
The study design of two phase 2 clinical studies examining a mutation-specific DMD therapy is presented in our poster from CNS 2019
The objectives and primary and secondary outcome measures of two interventional phase 2 studies: Study 045 (NCT03648827), a trial examining the percentage change in levels of dystrophin from baseline in ambulatory treatment-naïve boys with nmDMD after treatment with a mutation-specific DMD therapy for 40 weeks (completed 30 October 2020); and Study 046 (NCT03796637) investigating the quantitative change in levels of dystrophin in ambulatory nmDMD subjects as measured by electrochemiluminescence (ECL) receiving a mutation-specific DMD treatment for ≥9 months (completed 24 May 2019)
▼ 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.