Digital Biomarker Study Opens: Real-World Walking Assessment in DMD and SMA — Advancing Precision Monitoring for Rare Neuromuscular Disorders

References

1. 1.Columbia University. (2024). Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders. ClinicalTrials.gov. https://clinicaltrials.gov/study/NCT06839469
2. 2.Goemans, N., Klingels, K., van den Hauwe, M., Vuylsteke, M., Pauwels, P., De Cat, J., Molenaers, G., & Desloovere, K. (2016). Six-minute walk test: reference values and prediction equation in healthy boys aged 5 to 12 years. PLoS One, 11(1), e0147438. https://doi.org/10.1371/journal.pone.0147438
3. 3.McDonald, C. M., Henricson, E. K., Abresch, R. T., Duong, T., Joyce, N. C., Hu, F., Clemens, P. R., Hoffman, E. P., Cnaan, A., & Gordish-Dressman, H. (2018). The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: reliability, concurrent validity, and minimal clinically important differences. Muscle & Nerve, 57(6), 905-915. https://doi.org/10.1002/mus.26671
4. 4.Pane, M., Mazzone, E. S., Sivo, S., Sormani, M. P., Messina, S., D'Amico, A., Carlesi, A., Vita, G., Russo, M., Palermo, C., Berardinelli, A., Vasco, G., Tiziano, F. D., Bertini, E., & Mercuri, E. (2014). Long term natural history data in ambulant boys with Duchenne muscular dystrophy: 36-month changes. PLoS One, 9(10), e108205. https://doi.org/10.1371/journal.pone.0108205
5. 5.Servais, L., Deconinck, N., Moraux, A., Benali, M., Canal, A., Van Parys, F., Vereecke, E., Wittevrongel, L., Maes, B., & Desguerre, I. (2013). Innovative methods to assess upper limb strength and function in non-ambulant Duchenne patients. Neuromuscular Disorders, 23(2), 139-148. https://doi.org/10.1016/j.nmd.2013.06.014