Digital Endpoint Resource Guide: Neuromuscular Disorders

Developing drugs for neuromuscular disorders (NMDs) presents challenges due to suboptimal clinical endpoints for gauging efficacy. Regulatory and payer demands for patient-centered outcomes are rising. Sensor-based digital health technologies (DHTs) offer direct, objective daily life assessments, accelerating NMD drug development.

NMDs like ALS, DMD, and SMA are rare and marked by muscle weakness, significantly impairing daily activities. Traditional clinical assessments suffer from subjectivity and bias, causing longitudinal variability. Wearable DHTs provide continuous, objective function assessment, overcoming travel barriers for NMD patients in clinical trials. They serve as ideal functional endpoints in long-term trials or follow-ups, enabling remote data collection with minimal patient burden.

Learn more about how wearable DHTs offer both continuous, objective assessment of patient function in their natural environment, facilitating the detection of clinically significant effects, and reducing the need for patients with NMDs to travel to clinical sites, a major obstacle to study participation.