Registry Study Collecting Real-World Data on Assistive Devices, Medicines, and Healthcare in Patients with ALS, SMA, and Other Neurological Diseases

What this Trial Is About

Researchers are collecting real-world data on the use of assistive devices, medicines, and other healthcare measures in patients with Amyotrophic Lateral Sclerosis ALS, Spinal Muscular Atrophy SMA, and other neurological diseases. The study aims to understand clinical practice by gathering patient-reported outcomes and evidence from daily healthcare. It includes information on clinical characteristics, medical history, and treatment satisfaction. The study collects data on various treatments and supports such as ventilation therapy, nutrition support, and innovative drugs like Tofersen, Nusinersen, and Risdiplam. It also gathers information on symptomatic drugs and genetic markers relevant to these neurological conditions. Patient questionnaires and clinical scales are used to capture treatment effectiveness and patient expectations. Participants provide information over a long-term period, with the primary outcome focusing on systematic recording of neurological treatment and patient evaluations via specific questionnaires for up to 10 years. Data collected include biomarker levels, genetic mutation status, and clinical assessment scales like the ALS Functional Rating Scale-Revised ALSFRS-R. This observational registry study involves no interventional treatments and aims to monitor healthcare use and patient experiences comprehensively.

Registry Study of Assistive Devices, Medicines and Healthcare Measures in ALS, SMA and Other Neurological Diseases.