Amyotrophic Lateral Sclerosis (ALS)
from OrphaChat — a Rare Disease Podcast Hosted by RCH
Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative condition characterised by the loss of motor neurons, leading to progressive paralysis and respiratory failure. These sources detail diagnostic frameworks, such as the Gold Coast and Awaji criteria, alongside the emerging use of neurofilament light chain as a critical biomarker for monitoring disease progression. While established medications like Riluzole provide modest survival benefits, newer therapies such as Qalsody and investigational drugs like ATH-1105 target specific genetic mutations and toxic protein aggregations. Multidisciplinary care remains essential, focusing on nutritional support, respiratory interventions, and the integration of patient preferences to improve quality of life. Furthermore, epidemiological data predicts a significant rise in global prevalence by 2030, necessitating urgent reform in clinical trial design and public health resource allocation.
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