Home | Neuromuscular | Duchenne muscular dystrophy (DMD) | Publications | Publication: Safety and effectiveness of ataluren: Comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

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

This research article, published in the Journal of Comparative Effectiveness Research, outlines the safety and effectiveness of a mutation-specific Duchenne muscular dystrophy (DMD) therapy in two propensity score-matched DMD patient populations 

  • Review the safety of a mutation-specific DMD therapy in patients in the Strategic Targeting of Registries and International Database of Excellence (STRIDE) Registry (interim cut-off date: 9 July 2018)
  • View the effectiveness of this treatment, assessed by comparing outcomes in treated patients with those in a propensity score-matched DMD population from the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), who were comparable in established predictors of disease progression
  • Age at first symptoms as a measure of disease progression and age at loss of ambulation as a measure of treatment benefit are discussed

Mercuri E, Muntoni F, Nascimento Osorio A et al. J Comp Eff Res. 2020;9(5):341–360

Once registered, you will be able to view the abstract and be provided the DOI and PubMed links for this publication.

Register now to unlock the content

Register now to access the content on this page

Already registered?
If not, register below

Registration
  • This Website is intended only for healthcare professionals (“Website“), is operated by PTC Therapeutics. (together with its affiliates “PTC”). In order to access the restricted content on this Website:

    *
    *
  • PTC will delete immediately the information provided if we cannot confirm you are a healthcare professional.
    Otherwise, in case of successful verification, PTC will retain the information provided for a period of two (2) years following your registration (i) only for the purposes of providing you access to this Website; and/or (ii) in case you signed up to receive PTC updates.

Providing your personal data and your consent is voluntary, and you have the right to withdraw your consent at any time, without giving any reasons. Please consult our Privacy Statement for more information on the processing of your personal data.
For any inquiry relating to the processing of your personal data or if you want to exercise your privacy related rights or withdraw your consent, you can contact PTC’s Privacy Office at: Dataprivacy@ptcbio.com.

You can unsubscribe from updates at any time when receiving an email from PTC.

US-DMD-0263 | April 2023
Sign in or register to access exclusive content on this site 
  REGISTER

Welcome to MEDhub

A website for healthcare professionals, provided by PTC Therapeutics

This educational website provides you with the latest scientific data and expert
insights on the diagnosis and management of a range of rare diseases across the neuromuscular system, central nervous system, and other areas

This content is protected. To view it please enter your email address below:

Register here to access the content on the site
US-CORP-0330 | July 2022

COMING SOON

The content you are trying to access is not currently available.

We will be updating the MEDhub site regularly to provide you with up-to-date, insightful expert-led content. To be notified when new additions become available, register now.