At Cure SMA 2026, several presentations showcased some emerging clinical outcome measures for spinal muscular atrophy (SMA), including a refined patient-reported test, a practical functional tool, and an artificial intelligence (AI)–powered wearable gait-monitoring device. Developed for use in both clinical and research settings, these innovations aim to improve the assessment of daily function and disease burden in patients living with SMA.

Following these presentations, featured expert John Bandsema, MD, was interviewed by Conference Reporter Associate Editor-in-Chief Sandra Ramadani, PhD. Clinical perspectives from Dr Brandsema on these findings are presented here.

One of the reasons SMA research has been so successful is because of the innovative work of the SMA evaluator community worldwide. We welcome new approaches to assessment because no scale is perfect. We are always striving to do better when it comes to precisely understanding a patient’s disease burden and how it evolves over time.

At Cure SMA 2026, Maria Fragala-Pinkham, PT, DPT, DSc, presented a Rasch analysis of the Spinal Muscular Atrophy Person-Reported Outcome (SMA-PRO) daily activities domain, detailing how the measure performs in children and adults with SMA (abstract 14). The SMA-PRO and other patient-reported outcome measures (PROMs) are important because they bring a day-to-day “how does this actually affect the patient” spin on the symptoms we are trying to assess in clinic. In recent years, PROMs such as the SMA Experienced Fatigability Rating Tool (SMA EFFORT) and the Pediatric Evaluation of Disability Inventory Computer Adaptive Test (PEDI-CAT; Pearson Assessments US) have really started to gain traction in clinical trials and clinical practice. Nonetheless, these scales can be challenging to administer when they are too lengthy, have to be administered too frequently, or are not relevant to the patient’s daily life.

Furthermore, PROMs can help us objectively assess how patients are doing over time, but we have to put the test results in the context of a patient’s daily life. For example, maybe the patient was having a really busy week at the time of the assessment, and they happened to perform worse on the test than they typically would have. We have to take this kind of functional variability, as well as the patient’s mood at the time of testing, into account. These factors can all impact PROs.

In another study presented at Cure SMA 2026, Joelle Williamson Clark, MPH, discussed work on the Shuttle Squat Test, a practical tool designed to assess endurance and proximal leg muscle strength in individuals with SMA (abstract 13). I think that the Shuttle Squat Test is an attempt to make walking tests more feasible in the average busy clinic. Currently, the 6-Minute Walk Test (6MWT) is the standard test used in this setting, but it assumes that the clinician has 30 meters of space and 6 minutes available to them to perform it, which is not always the case. The Shuttle Squat Test is also something that patients seem to participate in fairly well. They are often willing to give it a try. However, the test seems to need further study on normative values and whether there are other aspects of how patients perform the test that need to be considered.

Also at this year’s Cure SMA conference, Kai-Chun Liu, PhD, described work on AI-Sole, an AI-powered wearable device for the long-term real-world monitoring of spatiotemporal and kinetic gait patterns in individuals with SMA (abstract 15). The work presented is preliminary, and it seems that AI-Sole would need to be used properly by the patient to ensure that the data collected are real and are not being overanalyzed.

I now use AI in my practice a bit too because I think that it offers many benefits to clinicians, including improved efficiency and ability to focus clearly on large amounts of data. However, we still need that human “piece” of looking over data for accuracy and drawing appropriate conclusions. So, I am not against AI helping us in the research and clinical worlds; I just think that it should be done very carefully and with adequate checks and balances.