DMD On-Demand Hub
All the 
TranslarnaTM (ataluren) webinars in one place. Catch up on the ones you have missed or revisit ones you have enjoyed.
Product-related information is discussed.
Exploring totality of evidence for a treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD): From clinical trial to real-world experience
Join Dr Dmitry Vlodavets, Professor Marika Pane, Dr Carolina Fischinger and Dr Flávia Nardes as they provide an up-to-date overview of the totality of evidence supporting Translarna for the treatment of nmDMD.
This is a recording of our live webinar, “Exploring totality of evidence for a treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD): From clinical trial to real-world experience”.
Navigating the changing disease landscape: Continuity of care in nonsense mutation Duchenne muscular dystrophy (nmDMD)
This is a recording of our live webinar, “Navigating the changing disease landscape: Continuity of care in nmDMD”.
Navigating the changing landscape: Continuity of care in Duchenne muscular dystrophy
Join Professor Marika Pane and Professor Craig McDonald as they discuss how best to manage DMD patients over the full disease continuum and provide an overview of the latest data from a Phase 3, placebo-controlled trial and an ongoing real-world evidence study in nonsense mutation DMD.
This is a recording of our live webinar, “DMD Continuum: Latest insights in DMD management.”
Delaying nmDMD milestones – preserving function and improving patient outcomes with Translarna
Watch Luca Bello and Craig McDonald as they explore slowing disease progression with Translarna, examine efficacy outcomes and real world evidence and discuss the value of functional assessments as prognostic indicators in nonsense mutation DMD.
This is a recording of our live webinar “Delaying nmDMD milestones – preserving function and improving patient outcomes with Translarna”.
DMD Continuum: From early intervention to adult care transition
This is a recording of our live webinar “The DMD Continuum: From early intervention to adult care transition”.
Safety and effectiveness results of a mutation-specific therapy for DMD in a real-world setting
This is a recording of our live webinar “Individualising DMD: Real-world management of a mutation-specific DMD”.
Translarna (ataluren): Global therapeutic indications
Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older in the European Member States and Iceland, Liechtenstein, Norway, Great Britain, Northern Ireland, Kazakhstan, Israel, Republic of Korea, Belarus, Russia, Brazil, Peru, Chile, Macedonia, Uruguay, and Serbia, and aged 5 years and older in the Kingdom of Saudi Arabia and Ukraine (under special state registration). In Brazil, the indication is specific to male paediatric patients. The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing (Translarna Summary of Product Characteristics (SmPC) for respective countries). Translarna received a conditional marketing authorisation in the European Member States and Iceland, Liechtenstein, Norway, Great Britain, and Northern Ireland.
Registration conditions differ internationally, always consult Prescribing Information and/or Summary of Products Characteristics before prescribing any product. For the EU Translarna Summary of Product Characteristics, please click here.
This medicinal product 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 pharmacovigilance@ptcbio.com.
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Copyright © 2024 PTC Therapeutics. All rights reserved. Privacy Statement | Cookie Statement
GL-TRNS-0737 | January 2024
The website and webinars are organised and funded by PTC Therapeutics, Inc. for healthcare professionals only. Trademarks, registered or otherwise, are the property of their respective owner(s).
Copyright © 2024 PTC Therapeutics. All rights reserved. Privacy Statement | Cookie Statement
GL-TRNS-0737 | January 2024
