cTAP Announces Novel Prognostic Score Developed for Duchenne Muscular Dystrophy Patients Offers Improved Prediction of Loss of Ambulation
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 nods and can better inform clinical decision-making and research than previously reported models. Its publication represents the most recent addition to cTAP’s suite of open access prognostic models and insights developed to support improved trial design and disease state understanding in DMD. “This new prognostic score represents a significant advancement in managing Duchenne muscular dystrophy,” said Craig McDonald, MD, Professor, University of California at Davis Medical Center. “By providing clinicians with a simple yet powerful tool to predict time to loss of ambulation, we can better counsel patients and families, optimize trial design, and ultimately improve the quality of care for individuals living with Duchenne.”
The prognostic score was developed using data from over 600 boys with DMD from natural history databases and clinical trial placebo arms. Researchers used classification and regression tree models, prioritizing easily obtainable functional measures, rise from floor and the 10-meter walk/run. These predictors were used to classify patients into five distinct risk groups.
The loss of ambulation remains one of the most significant challenges faced by individuals living with Duchenne and their families,” said Debra Miller, founder and CEO of CureDuchenne, a founding patient advocacy partner and provider of initial seed funding to cTAP. “This novel prognostic score will help improve research for treatments that aim to stop or delay Duchenne’s relentless progression and support clinicians to better assist families in planning ahead to navigate this transition. We are eager to see the score put into use, and we are grateful to have played a part by contributing critical patient data from a prospectively defined natural history study to help develop the model. This cTAP effort is an example of the importance of partnerships that bring patient organizations, academics, and industry together to solve problems.”
The study demonstrated that this prognostic score effectively stratified patients into well-separated risk groups with median time to LoA > 4 years in the lowest risk group and 0.9 years in the highest-risk group. Crucially, the score was validated using an independent dataset, confirming its robustness and generalizability. The developed score outperformed existing classifications based on individual functional tests, offering a more accurate prediction of LoA timelines.
The utility of this prognostic score extends to clinical practice, enabling healthcare providers to offer more personalized anticipatory guidance on mobility devices, home modifications, and psychosocial support. In clinical trials, it can enhance patient stratification and potentially improve the efficiency of research by identifying subgroups with similar prognostic trajectories.
The publication is available with open access below: https://www.neurology.org/doi/10.1212/WNL.0000000000214705
Duchenne muscular dystrophy is a severe, X-linked genetic disorder affecting 1:3500-1:6000 live births. It is characterized by progressive muscle degeneration and loss of motor function, eventually leading to early death due to cardiopulmonary failure.
cTAP is a multinational, multistakeholder nonprofit organization dedicated to advancing clinical trial design, analysis, and patient data resources in rare neuromuscular diseases. Leveraging expansive natural history and placebo datasets, a highly engaged network of world-class experts, and a proven track record of success in collaborative problem-solving, cTAP develops evidence-based advancements with real-world impact. cTAP’s work in Duchenne muscular dystrophy underpins improved clinical trial design, analysis and interpretation, superior matching to external controls, and stronger evidence packages for regulators and health authorities. cTAP’s signature approach to catalyzing collaboration around patient data to accelerate research progress holds promise for rare, heterogeneous diseases and patient communities worldwide. Visit ctap-nmd.org to connect with cTAP or learn more.
Susan J. Ward, Ph.D.
Founder and Executive Director
Collaborative Trajectory Analysis Project (cTAP)
[email protected]
+1 617-448-2617
Novel prognostic score developed for patients with Duchenne Muscular Dystrophy offers improved prediction of loss of ambulation.