Gabriella Casagrande

Background: Assistive technology offers an important means of compensating for lost upper extremity function in adults with progressive neuromuscular diseases (NMD), enabling participation in daily activities, supporting independence, and promoting quality of life. However, the range of available technologies and the evidence supporting their use have not been comprehensively summarized. Aim: The aim of this scoping review was to identify and characterize upper extremity assistive technologies tested in adults with NMD and to summarize the current evidence regarding their functional applications and clinical outcomes. Materials and Methods: Electronic searches for published and unpublished literature were conducted using MEDLINE, Embase.com, Web of Science, Cochrane Central, and IEEE Xplore. The search strategy incorporated controlled vocabulary and free‐text synonyms for the concepts of upper extremity, rehabilitation, selected progressive neurodegenerative diseases, and assistive equipment. Following title/abstract and full‐text screening, studies evaluating assistive devices tested on adults with NMD during functional task performance were included. Results: After screening 2289 articles, 27 studies met the inclusion criteria. The studies collectively demonstrate the potential benefits and diverse range of assistive devices available to support upper extremity function. These devices ranged from low‐tech solutions, such as static mobile arm supports and fabricated splints, to high‐tech devices, including dynamic mobile arm supports, robotic systems, exoskeletons, and brain–computer interface systems. However, most studies were feasibility or case studies that primarily demonstrated proof of concept, with limited evidence regarding long‐term effectiveness, functional outcomes, or quality of life. Discussion: The findings illustrate the rapidly evolving landscape of upper extremity assistive devices for adults with NMD and their potential to improve functional performance, while highlighting the need for prospective studies that assess meaningful improvements in function, participation, and quality of life. Conclusions: As advances in disease‐modifying therapies extend survival and preserve function for individuals with NMD, interdisciplinary collaboration among engineers, clinicians, therapists, individuals with NMD, caregivers, and regulators will be essential to develop, evaluate, and implement assistive technologies that meet users' evolving needs.

Background: Accessing multidisciplinary care poses challenges for people living with amyotrophic lateral sclerosis (ALS) due to mobility issues. As ALS care rarely requires hospital‐based technology, most care is available through home visits. The Daniella Lipper ALS House Call Program (HCP) at Massachusetts General Hospital (MGH), launched in 2017 in collaboration with Compassionate Care ALS, has pioneered home‐based ALS care in Eastern Massachusetts. Methods: A retrospective chart review of ALS and primary lateral sclerosis (PLS) patients enrolled in the HCP at MGH was conducted. Data on demographics, visit details, and procedures performed during home visits were collected from electronic health records for patients seen from January 2024 to December 2024. Results: In 2024, the ALS HCP conducted 959 visits for 142 patients (average age: 68 years, range: 36–93; 47.9% female). Of these patients, 137 (96.5%) were diagnosed with ALS and 5 (3.5%) with PLS. Notably, 61 patients (43%) received care exclusively at home. Key interventions included 44 gastrostomy tube exchanges and 59 respiratory assessments, both of which significantly reduced hospital visits. The average distance traveled by the care team was 30.32 miles per visit. Conclusions: The Daniella Lipper ALS HCP at MGH brings ALS expertise into the patient’s home, minimizing travel burdens and ensuring continuity of care. The program illustrates the feasibility and impact of home‐based ALS care, suggesting potential for broader implementation across the nation. Development will focus on expanding services, such as tracheostomy changes in the homes, and on creating sustainable models for similar initiatives.

Introduction/Aims: Expanded access protocols (EAPs) allow individuals ineligible for clinical trials to receive investigational products. EAP data can be collected in parallel to randomized clinical trials (RCTs) and serve as a source of evidence in clinical practice. Here, we present the results of a National Institutes of Health (NIH)‐funded EAP for amyotrophic lateral sclerosis (ALS). Methods: Participants received trehalose, a drug studied in a parallel RCT, for up to 24 weeks; clinical and biomarker data were collected throughout the study. Results: Seventy participants were enrolled at 20 study centers across the United States. Treatment with trehalose did not affect the levels of neurofilament light chain [estimated flat slope per month was −0.005, SE = 0.0078; 95% CI (−0.021, 0.011)] or disease progression [estimated least square mean change of the ALS Functional Rating Scale—Revised total score and slow vital capacity (percent predicted) from baseline to Week 24 were −5.6 (0.67); 95% CI (−7.0, −4.3) and −4.53 (4.308); 95% CI (−13.55, 4.48)], respectively. No unexpected treatment‐related risks were identified. Serious adverse events were deemed not related to trehalose (20 occurrences in 13 [18.6%] participants with eight deaths). Discussion: This EAP establishes a framework for implementing multi‐center EAPs that complement data collected from RCTs. Additional NIH‐funded EAPs are currently underway. Data and additional serum samples collected in this study are available to the research community for further study. Trial Registration: ClinicalTrials.gov: NCT05597436