cTAP Announces Novel Prognostic Score Developed for Duchenne Muscular Dystrophy Patients Offers Improved Prediction of Loss of Ambulation

CAMBRIDGE, Mass.--(BUSINESS WIRE)--The Collaborative Trajectory Analysis Project (cTAP) announces the publication of a novel, validated prognostic score for predicting the loss of ambulation (LoA) in patients with Duchenne muscular dystrophy (DMD). According to research published May 26, 2026, in Neurology®, the medical journal of the American Academy of Neurology, this simple, easy-to-use score, based on two common functional assessments, rise from floor, and 10-meter walk/run, provides a more refined risk stratification and can better inform...

17

Aug 2026

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A Validated Prognostic Score for Time to Loss of Ambulation in Patients With Duchenne Muscular Dystrophy

Development of a prognostic score for time to loss of ambulation (LoA) may help guide treatment of patients with Duchenne muscular dystrophy (DMD). Information from clinical data sources was used to create a prognostic score for LoA for use in practice. Read Full Abstract See Full Publication in Neurology

26

May 2026

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Meaningful changes in motor function in Duchenne muscular dystrophy (DMD): A multi-center study

Evaluations of treatment efficacy in Duchenne muscular dystrophy (DMD), a rare genetic disease that results in progressive muscle wasting, require an understanding of the 'meaningfulness' of changes in functional measures. We estimated the minimal detectable change (MDC) for selected motor function measures in ambulatory DMD, i.e., the minimal degree of measured change needed to be confident that true underlying change has occurred rather than transient variation or measurement error. Read Full Abstract See Full Publication...

15

Aug 2024

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Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials

This study examined functional trajectories of subjects during the transition phase between ambulatory and non-ambulatory Duchenne muscular dystrophy (DMD) to inform clinical trial designs for new therapeutics. Ambulatory, pulmonary, and upper limb function leading up to loss of ambulation (LoA) and non-ambulatory measures following LoA were quantified; time ordering of pulmonary and upper limb milestones relative to LoA were determined; and the 10-second time threshold for 10-meter walk/run (10MWR) as a marker of approaching LOA...

15

Aug 2024

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New study may help more DMD patients be included in clinical trials

Muscular Dystrophy News Today covered the press release for cTAP’s recent publication “Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials.” Muscular Dystrophy News Today is daily digital news journal that seeks to give MD patients and their support systems access to the latest science and research news pertaining to the disease. View Full Article The publication was also covered on The American Journal of Managed Care...

15

Aug 2024

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Press release: cTAP Study Indicates DMD Clinical Trial Enrollment Criteria Should Expand Beyond Ambulatory Status

Jul 24, 2024 - Study co-funded by cTAP and CureDuchenne highlights the need for more specific enrollment criteria that could increase patient participation and drive more comprehensive therapeutic evaluation. Read the full press release on PR Newswire Read the full scientific article in PLoS ONE

15

Aug 2024

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Minimal detectable changes in functional measures in Duchenne muscular dystrophy: a study of multiple centers, networks and trial arms

Minimal detectable changes in functional measures in Duchenne muscular dystrophy: a study of multiple centers, networks and trial arms The design of clinical trials and evaluations of treatment efficacy in DMD require an understanding of the meaningfulness of changes in functional measures. An important concept is the minimal detectable change (MDC), i.e., the minimal magnitude of measured change indicative of true changes due to acquisition or loss of motor function related to the disease, rather...

27

Dec 2023

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Prognostic factors for pulmonary milestones in Duchenne muscular dystrophy

Prognostic factors for pulmonary milestones in Duchenne muscular dystrophy Knowledge of prognostic factors for pulmonary outcomes in DMD serves a general understanding of natural history and can help facilitate externally controlled studies and the long-term evaluation of novel therapies. This study assessed prognostic factors for time to forced vital capacity (FVC) %-predicted < 80% and time to FVC %-predicted < 50% using Cox proportional hazards analyses. This poster was presented at WMS 2021. Authors: N....

27

Dec 2023

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Functional trajectories of upper limb and pulmonary function before and after loss of ambulation in Duchenne muscular dystrophy

Functional trajectories of upper limb and pulmonary function before and after loss of ambulation in Duchenne muscular dystrophy Characterization of disease progression in DMD patients around loss of ambulation is needed to inform selection criteria and choice of endpoints in clinical trials including these patients. This study analyzed upper limb and pulmonary function in the years immediately before and after LoA (defined as inability to walk 10 meters). This poster was presented at WMS 2021....

27

Dec 2023

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Associations between dystrophin genotype and ambulatory outcomes in DMD: implications for trials of genotype-targeted therapies

Associations between dystrophin genotype and ambulatory outcomes in DMD: implications for trials of genotype-targeted therapies This study evaluated the suitability of genotypically unmatched controls in DMD by quantifying genotype effects on 1-year changes in North Star Ambulatory Assessment (NSAA) total score across data sources. This late-breaking poster was presented at WMS 2021. Authors: F. Muntoni, J. Signorovitch, G. Sajeev, H. Lane, M. Jenkins, I. Dieye, S. Ward, C. McDonald, N. Goemans, E. Niks, B. Wong,...

27

Dec 2023

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