This poster, presented at WMS 2022, focuses on the safety and efficacy results of Study 041, a phase 3 placebo-controlled trial of ataluren for patients with nonsense mutation Duchenne muscular dystrophy (nmDMD)
This poster, presented at WMS 2022, shows findings from a registry study investigating the effects of a mutation-specific treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD) on upper limb function
The best practice management and continuity of care in patients with Duchenne muscular dystrophy (DMD) was the focus of this symposium, presented at WMS 2022
This poster, presented at the 2022 14th EPNS Congress in Glasgow, discusses the functional outcomes of patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) receiving a mutation-specific DMD therapy in real-world practice versus a clinical trial setting
View an analysis of motor function in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) receiving ataluren in real-world practice versus a clinical trial setting in our poster presented at EPNS 2022
Presented at the 2022 AAN Annual Meeting in Seattle, USA, this poster shows results from a study investigating disease progression following treatment with different corticosteroids for Duchenne muscular dystrophy (DMD)
This poster, presented at the 2022 AAN Annual Meeting in Seattle, USA, presents the results of a comparison of corticosteroid treatments in non-ambulatory patients with DMD
View the key takeaways from our MDA 2022 Virtual Industry Forum, which summarizes the role of corticosteroids in the treatment of patients with Duchenne muscular dystrophy (DMD)
Watch our hybrid Industry Forum at MDA 2022, which discusses the latest clinical findings on the role of corticosteroid treatment in the management of Duchenne muscular dystrophy (DMD)
This poster, from the 2022 MDA Conference in Nashville, Tennessee, USA, presents the ambulatory outcomes observed following treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD) in the STRIDE Registry and Study 020, a Phase 3 clinical trial
This poster, presented at the 2022 MDA Conference in Nashville, Tennessee, USA, reports the results of a comparison of corticosteroid treatments on clinical outcomes in non-ambulatory DMD patients
This poster from the 2022 MDA Conference in Nashville, Tennessee, USA, presents results from a study comparing clinical outcomes in patients with Duchenne muscular dystrophy (DMD) by corticosteroid type within distinct patient subgroups
This poster, presented at the 2022 MDA Conference in Nashville, Tennessee, USA, shows the results of a study comparing 2 corticosteroids in delaying disease progression milestones in patients with DMD
This poster, presented at the 2022 MDA Conference in Nashville, Tennessee, USA, shows results from a propensity score-matched registry analysis, using data from the STRIDE Registry and CINRG Natural History Study, to determine the effect of a mutation-specific treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD) on pulmonary function
This poster, from the 2022 MDA Conference in Nashville, Tennessee, USA, compares the age at loss of ambulation in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) enrolled in the STRIDE Registry and CINRG Natural History Study
This poster, presented at the 2022 MDA Conference in Nashville, Tennessee, USA, provides safety data from a registry analysis of boys with nonsense-mutation Duchenne muscular dystrophy (nmDMD)
This poster, presented at the virtual 2021 AMCP Annual Meeting, shows findings from a study comparing the effects of treatment on time to LoA and pulmonary function decline in nmDMD
This poster, shown at the 2021 virtual AMCP Annual Meeting, presents results of a qualitative interview study investigating the impact on health-related quality of life (HRQoL) of nmDMD, and patients’ and caregivers’ experience of treatment
This poster, presented at the 2021 virtual AMCP Annual Meeting, shows the key findings of an investigation into the symptoms and impact of nmDMD at different stages of ambulation
This poster, presented at the 2021 virtual AMCP Annual Meeting, describes a literature review to establish core prognostic indicators for DMD, to help inform propensity score-matching models in studies
This poster, presented at the virtual 2021 MDA Conference, shows findings from a study comparing the effects of treatment on time to LoA and pulmonary function decline in nmDMD
This poster, presented at the 2021 virtual MDA Conference, shows the methodology and importance of propensity score-matching in clinical trials where random assignment to treatment is not possible
This poster, displayed at the 2021 virtual MDA Conference, shows findings from an investigation into the real-world reasons for switching between corticosteroids for the treatment of dystrophinopathies
This poster, presented at the ICNMD virtual meeting in 2021, shows findings from a registry study investigating the effects of a mutation-specific Duchenne muscular dystrophy (DMD) therapy on pulmonary function in patients with DMD
This poster, presented at the ICNMD virtual meeting in 2021, shows the results from a study evaluating efficacy of a mutation-specific DMD therapy
This poster, presented at the 2021 virtual WMS Annual Congress, presents a comparison of ambulatory outcomes following treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD) in a registry study and a Phase 3 clinical trial
This poster, from the 2021 virtual AAN meeting, presents findings of a review of real-world steroid switching in US patients with muscular dystrophies
This poster, from the 2021 virtual AAN meeting, provides results from a study evaluating the efficacy of a mutation-specific treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD)
This poster, from the virtual 2021 ICNMD meeting, shows the demographic and safety data for patients included in the STRIDE Registry population
This poster, presented at the 2021 virtual MDA Conference, presents findings from a literature review to determine core prognostic indicators for DMD, to help inform propensity score-matching models in studies
Watch our virtual symposium at ICNMD 2020, which discusses long-term real-world evidence (RWE) regarding ataluren use in patients with nonsense mutation DMD (nmDMD)
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Access PTC abstracts, infographics, posters and symposia presented at international meetings in the fields of pediatric neurology, cognitive neuroscience and movement disorders, such as the Muscular Dystrophy Association (MDA) Annual Clinical & Scientific Conference, the Child Neurological Society (CNS) Annual Meeting, the World Muscle Society (WMS) Annual Congress, and the European Paediatric Neurology Society (EPNS) Congress.
▼ 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 pharmacovigilance@ptcbio.com.
Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The European Summary of Product Characteristics for ataluren is available here. Please refer to your local country guidance for more information.
Deflazacort is a corticosteroid indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients 2 years of age and older. PTC Therapeutics is the Marketing Authorization Holder for deflazacort in the US only. Registration conditions differ internationally, and prescribing information may vary depending on local approval in each country. The US Prescribing Information for deflazacort is available here. Please refer to your local country guidance for more information.