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Press release: cTAP Study Indicates DMD Clinical Trial Enrollment Criteria Should Expand Beyond Ambulatory StatusPress release: cTAP Study Indicates DMD Clinical Trial Enrollment Criteria Should Expand Beyond Ambulatory StatusAugust 15, 2024 -
New study may help more DMD patients be included in clinical trialsNew study may help more DMD patients be included in clinical trialsAugust 15, 2024 -
Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trialsFunctional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trialsAugust 15, 2024
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Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center studyMeaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center studyAugust 15, 2024
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Beyond Traditional Placebo Controls in Rare Disease Clinical Trials: New cTAP Evidence Supports Patient-Centric AlternativesBeyond Traditional Placebo Controls in Rare Disease Clinical Trials: New cTAP Evidence Supports Patient-Centric AlternativesFebruary 8, 2023 -
DMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical TrialsDMD Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical TrialsFebruary 1, 2023
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Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controlsReal-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with external controlsApril 1, 2022 -
Poster: Predicting Trajectories of Ambulatory Function in Duchenne Muscular DystrophyPoster: Predicting Trajectories of Ambulatory Function in Duchenne Muscular DystrophyApril 6, 2022 -
Poster: A Concisely Recorded Ambulatory Assessment for Enhancing Real-World Outcomes Research in Duchenne Muscular Dystrophy: Development and ValidationPoster: A Concisely Recorded Ambulatory Assessment for Enhancing Real-World Outcomes Research in Duchenne Muscular Dystrophy: Development and ValidationApril 6, 2022
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A Concisely Recorded Ambulatory Assessment for Enhancing Real-World Outcomes Research in Duchenne Muscular Dystrophy: Development and ValidationA Concisely Recorded Ambulatory Assessment for Enhancing Real-World Outcomes Research in Duchenne Muscular Dystrophy: Development and ValidationDecember 27, 2023
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Consistency of changes in %-predicted forced vital capacity between real-world data and trial placebo arms in ambulatory Duchenne muscular dystrophyConsistency of changes in %-predicted forced vital capacity between real-world data and trial placebo arms in ambulatory Duchenne muscular dystrophyDecember 27, 2023
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Development and Evaluation of a Time to Event Endpoint for Clinical Trials in Duchenne Muscular Dystrophy (DMD)Development and Evaluation of a Time to Event Endpoint for Clinical Trials in Duchenne Muscular Dystrophy (DMD)December 27, 2023
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Genotype-unmatched controls are feasible for drug development in Duchenne Muscular Dystrophy (DMD)Genotype-unmatched controls are feasible for drug development in Duchenne Muscular Dystrophy (DMD)December 27, 2023
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Validation of a composite prognostic score for time to loss of ambulation in Duchenne Muscular DystrophyValidation of a composite prognostic score for time to loss of ambulation in Duchenne Muscular DystrophyDecember 27, 2023
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Accelerating clinical development of new therapeutics with patient data: evidence from the collaborative Trajectory Analysis Program (cTAP) in DMDAccelerating clinical development of new therapeutics with patient data: evidence from the collaborative Trajectory Analysis Program (cTAP) in DMDDecember 27, 2023
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The Problem with Clinical Trials in Duchenne—and What We Can Do About ItThe Problem with Clinical Trials in Duchenne—and What We Can Do About ItNovember 16, 2021 -
2021 WMS Late Breaking Poster - Genotype Study2021 WMS Late Breaking Poster - Genotype StudySeptember 24, 2021 -
Associations between dystrophin genotype and ambulatory outcomes in DMD: implications for trials of genotype-targeted therapiesAssociations between dystrophin genotype and ambulatory outcomes in DMD: implications for trials of genotype-targeted therapiesDecember 27, 2023
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Functional trajectories of upper limb and pulmonary function before and after loss of ambulation in Duchenne muscular dystrophyFunctional trajectories of upper limb and pulmonary function before and after loss of ambulation in Duchenne muscular dystrophyDecember 27, 2023
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Prognostic factors for pulmonary milestones in Duchenne muscular dystrophyPrognostic factors for pulmonary milestones in Duchenne muscular dystrophyDecember 27, 2023
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Minimal detectable changes in functional measures in Duchenne muscular dystrophy: a study of multiple centers, networks and trial armsMinimal detectable changes in functional measures in Duchenne muscular dystrophy: a study of multiple centers, networks and trial armsDecember 27, 2023
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cTAP at the 2021 RE(ACT) CONGRESS and IRDIRC ConferencecTAP at the 2021 RE(ACT) CONGRESS and IRDIRC ConferenceJanuary 15, 2021 -
cTAP at the 2021 PPMD Duchenne Drug Development RoundtablecTAP at the 2021 PPMD Duchenne Drug Development RoundtableDecember 27, 2023 -
cTAP Study Shows Real-World, Natural History Data Comparable to Placebo Data from Clinical Trials in Duchenne Muscular DystrophycTAP Study Shows Real-World, Natural History Data Comparable to Placebo Data from Clinical Trials in Duchenne Muscular DystrophySeptember 8, 2020
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Deflazacort vs prednisone treatment for Duchenne muscular dystrophyDeflazacort vs prednisone treatment for Duchenne muscular dystrophyJanuary 10, 2020 -
A prognostic model for 1 year change in 4SCA prognostic model for 1 year change in 4SCJune 23, 2020 -
Suitability of external controls for drug evaluation in Duchenne muscular dystrophySuitability of external controls for drug evaluation in Duchenne muscular dystrophySeptember 8, 2020
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Real-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophyReal-world outcomes of long-term prednisone and deflazacort use in patients with Duchenne muscular dystrophyJanuary 10, 2020 -
Prognostic factors for loss of ability to rise from supine in DMDPrognostic factors for loss of ability to rise from supine in DMDSeptember 28, 2020 -
cTAP at the 2020 PPMD Clinical Care Conference (Virtual Congress)cTAP at the 2020 PPMD Clinical Care Conference (Virtual Congress)December 27, 2023
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cTAP Publishes Comprehensive Analysis of NSAAcTAP Publishes Comprehensive Analysis of NSAASeptember 26, 2019 -
Nature Biotechnology: Their Lives in Their HandsNature Biotechnology: Their Lives in Their HandsNovember 6, 2019 -
Categorising trajectories and individual item changes of the NSAA in patients with DMDCategorising trajectories and individual item changes of the NSAA in patients with DMDSeptember 3, 2019
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2019 EAN Abstract - Phase 1b/2 Study of RG62062019 EAN Abstract - Phase 1b/2 Study of RG6206September 1, 2019 -
2019 EU ISPOR Abstract - NSAA consistency analysis2019 EU ISPOR Abstract - NSAA consistency analysisJune 12, 2019 -
2019 EU ISPOR Abstract - Prognostic Model for Loss of Walking in DMD2019 EU ISPOR Abstract - Prognostic Model for Loss of Walking in DMDJune 5, 2019
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2019 JSCN Abstract - Phase 1b/2 Study of RG62062019 JSCN Abstract - Phase 1b/2 Study of RG6206May 31, 2019 -
2019 MDA Abstract - The Collaborative Trajectory Analysis Project (cTAP)2019 MDA Abstract - The Collaborative Trajectory Analysis Project (cTAP)April 13, 2019 -
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2019 Myology Abstract - Changes in 4SC over Two Years in DMD2019 Myology Abstract - Changes in 4SC over Two Years in DMDMarch 25, 2019 -
2019 PTC WMS Abstract - Steroid Use and DMD2019 PTC WMS Abstract - Steroid Use and DMDApril 3, 2019
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2019 WMS Abstract - Estimating Changes in NSAA and 4SC in DMD2019 WMS Abstract - Estimating Changes in NSAA and 4SC in DMDApril 2, 2019
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2019 WMS Abstract - Lean Body Mass and Motor Function in DMD Patients2019 WMS Abstract - Lean Body Mass and Motor Function in DMD PatientsApril 2, 2019
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2019 WMS Abstract - 48-Week Changes in NSAA2019 WMS Abstract - 48-Week Changes in NSAAApril 2, 2019
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2019 WMS Abstract - Prognostic score for loss of walking ability in DMD2019 WMS Abstract - Prognostic score for loss of walking ability in DMDApril 2, 2019
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cTAP to Present Late-breaking Results at the World Muscle Society CongresscTAP to Present Late-breaking Results at the World Muscle Society CongressOctober 1, 2018
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cTAP to Present at Annual Biopharmaceutical ConferencecTAP to Present at Annual Biopharmaceutical ConferenceJune 24, 2018 -
Moneyball Medicine: Thriving in the new data-driven healthcare marketMoneyball Medicine: Thriving in the new data-driven healthcare marketOctober 17, 2018 -
cTAP Partner Wins ISPOR Award on Duchenne Muscular DystrophycTAP Partner Wins ISPOR Award on Duchenne Muscular DystrophyDecember 5, 2018 -
2018 Action Duchenne abstract - Consistency of 1-year change in 6MWD2018 Action Duchenne abstract - Consistency of 1-year change in 6MWDAugust 30, 2018 -
2018 Action Duchenne abstract - Longitudinal analyses of 4SC velocity in Leuven2018 Action Duchenne abstract - Longitudinal analyses of 4SC velocity in LeuvenOctober 17, 2018
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2018 Action Duchenne abstract - MCID of 4SC and NSAA in CCHMC2018 Action Duchenne abstract - MCID of 4SC and NSAA in CCHMCAugust 27, 2018
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Action Duchenne International Conference 2018 Abstract - Phase 1b/2 study of the anti-myostatin adnectin RG6206Action Duchenne International Conference 2018 Abstract - Phase 1b/2 study of the anti-myostatin adnectin RG6206August 29, 2018
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2018 ISPOR EU Abstract - Ambulatory Function in Duchenne Muscular Dystrophy2018 ISPOR EU Abstract - Ambulatory Function in Duchenne Muscular DystrophyOctober 17, 2018
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WMS 2018 Abstract - 4SC Velocity Prognostic ModelWMS 2018 Abstract - 4SC Velocity Prognostic ModelOctober 2, 2018
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WMS 2018 Abstract - Minimal detectable change in NSAAWMS 2018 Abstract - Minimal detectable change in NSAAOctober 3, 2018
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cTAP at the 2018 Action Duchenne ConferencecTAP at the 2018 Action Duchenne ConferenceNovember 9, 2018
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cTAP Presentation at the 2018 ASA Biopharmaceutical Section Regulatory-Industry Statistics WorkshopcTAP Presentation at the 2018 ASA Biopharmaceutical Section Regulatory-Industry Statistics WorkshopSeptember 12, 2018 -
Marathon Pharmaceuticals and Italfarmaco Join cTAPMarathon Pharmaceuticals and Italfarmaco Join cTAPFebruary 7, 2017 -
Modern Solutions to Recurring Roadblocks in Clinical TestingModern Solutions to Recurring Roadblocks in Clinical TestingMarch 6, 2017 -
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Summit Therapeutics Joins cTAP to Support the Development of Potential DMD TreatmentsSummit Therapeutics Joins cTAP to Support the Development of Potential DMD TreatmentsSeptember 29, 2017 -
2017 IRDiRC Abstract - Translating Natural History into Clinical Trial Design2017 IRDiRC Abstract - Translating Natural History into Clinical Trial DesignFebruary 3, 2017 -
2017 WMS Abstract - Clustering Trajectories of Ambulatory Function2017 WMS Abstract - Clustering Trajectories of Ambulatory FunctionOctober 3, 2017
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2017 WMS Abstract - Consistency between natural history and clinical trial placebo2017 WMS Abstract - Consistency between natural history and clinical trial placeboOctober 3, 2017
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2017 WMS Abstract - The Collaborative Trajectory Analysis Collaboration2017 WMS Abstract - The Collaborative Trajectory Analysis CollaborationOctober 3, 2017
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2017 WMS Abstract - Validation of a prognostic score for changes in 6MWD2017 WMS Abstract - Validation of a prognostic score for changes in 6MWDOctober 3, 2017
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Non-Profits, Pharmaceutical Companies and Noted Clinicians Collaborate to Launch cTAPNon-Profits, Pharmaceutical Companies and Noted Clinicians Collaborate to Launch cTAPOctober 25, 2016 -
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Nontraditional Approaches to Big Data Analyses: A Case Study in Rare DiseaseNontraditional Approaches to Big Data Analyses: A Case Study in Rare DiseaseDecember 1, 2016 -
Categorizing natural history trajectories of ambulatory functionCategorizing natural history trajectories of ambulatory functionMay 24, 2016 -
Individualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular DystrophyIndividualized Prediction of Changes in 6-Minute Walk Distance for Patients with Duchenne Muscular DystrophyOctober 13, 2016 -
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cTAP at the World Congress Orphan Disease Conference 2015cTAP at the World Congress Orphan Disease Conference 2015November 11, 2015