Sodium phenylbutyrate and taurursodiol have been found to reduce neuronal death in experimental models. The efficacy and safety of a combination of the two compounds in persons with amyotrophic lateral sclerosis (ALS) are not known.

An orally administered, fixed‐dose coformulation of sodium phenylbutyrate‐taurursodiol (PB‐TURSO) significantly slowed functional decline in a randomized, placebo‐controlled, phase 2 trial in ALS (CENTAUR). Herein we report results of a long‐term survival analysis of participants in CENTAUR. In CENTAUR, adults with ALS were randomized 2:1 to PB‐TURSO or placebo. Participants completing the 6‐month (24‐week) randomized phase were eligible to receive PB‐TURSO in the open‐label extension. An all‐cause mortality analysis (35‐month maximum follow‐up post‐randomization) incorporated all randomized participants. Participants and site investigators were blinded to treatment assignments through the duration of follow‐up of this analysis. Vital status was obtained for 135 of 137 participants originally randomized in CENTAUR. Median overall survival was 25.0 months among participants originally randomized to PB‐TURSO and 18.5 months among those originally randomized to placebo (hazard ratio, 0.56; 95% confidence interval, 0.34‐0.92; P = .023). Initiation of PB‐TURSO treatment at baseline resulted in a 6.5‐month longer median survival as compared with placebo. Combined with results from CENTAUR, these results suggest that PB‐TURSO has both functional and survival benefits in ALS.

Despite promising results in the rehabilitation field, it remains unclear whether upper limb robotic wearables, e.g., for people with physical impairments resulting from neurodegenerative disease, can be made portable and suitable for everyday use. We present a lightweight, fully portable, textile-based, soft inflatable wearable robot for shoulder elevation assistance that provides dynamic active support to the upper limbs. The technology is mechanically transparent when unpowered, can quantitatively assess free movement of the user, and adds only 150 grams of weight to each upper limb. In 10 individuals with amyotrophic lateral sclerosis (ALS) with different degrees of neuromuscular impairment, we demonstrated immediate improvement in the active range of motion and compensation for continuing physical deterioration in two individuals with ALS over 6 months. Along with improvements in movement, we show that this robotic wearable can improve functional activity without any training, restoring performance of basic activities of daily living. In addition, a reduction in shoulder muscle activity and perceived muscular exertion, coupled with increased endurance for holding objects, highlight the potential of this device to mitigate the impact of muscular fatigue for patients with ALS. These results represent a further step toward everyday use of assistive, soft, robotic wearables for the upper limbs.