Management and therapeutic perspectives in amyotrophic lateral sclerosis

Abstract

Amyotrophic lateral sclerosis (ALS) is a fatal progressive neurodegenerative disorder affecting both upper and lower motor neurons. Despite much research and effort, no clear insights into a unifying hypothesis for the pathogenesis has so far emerged for this disease. Areas covered: We review the main pathophysiological hypotheses and the potential therapeutic targets in ALS, as well as the management of these patients (in order to improve their survival and quality of life). Expert commentary: ALS is a complex neurodegenerative disease, these days considered as a multisystem disorder with predominant motor symptoms (and various clinical forms). Further comprehension of the pathophysiology of this disease is required, although pathophysiological mechanisms (such as TDP-43) show promise in the search for new therapies. There is still no curative treatment for ALS, but the emergence of multidisciplinary specialized ALS clinics has increased both the quality of life and the survival of these patients.

RNA-binding proteins with prion-like domains in health and disease

“…ALS pathology also extends to the peripheral nervous system [5,10]. Our lack of understanding of the mechanisms and risk factors governing the development and progression of neurodegenerative diseases has largely precluded the development of disease-reversing therapeutics [4,11,12]. Symptomatic treatments are available for PD and AD, but the efficacy of these can be modest or limited by problematic side effects, and they do not address the root cause of disease [12,13].…”

Rehabilitation bei Amyotropher Lateralsklerose

“…Ebenso sinnvoll wie die Etablierung rehabilitativer Interventionen in multidiszip linären ALSKliniken, die in der Regel an Krankenhäusern der höchsten Versorgungsstufe angesiedelt sind, sind speziell auf ALSPatienten ausgerichteten stationäre und ambulante Re habilitationsmaßnahmen. Schließlich profitieren ALS-Patien ten von rehabilitativen Interventionen durch eine Reduktion der Symptomlast und eine Verbesserung der Lebensqualität [111,112], ihre Überlebenszeit wird überdies verlängert [97][98][99][100]. Sowohl eine beatmungsmedizinische als auch eine palliativmedizinische Expertise sind für den Erfolg der Rehabilitation allerdings wesentlich.…”

RNA-Targeted Therapies and Amyotrophic Lateral Sclerosis

“…Over the past decade, compelling clinical, imaging, and neuropathological data have provided evidence that the disease process involves not only motor neurons but also non-neuronal cells such as astrocytes and microglia [ 1 ]. ALS is no longer considered a strictly motor degenerative disorder because non-motor symptoms, including cognitive dysfunction, progressive supranuclear palsy-type eye movements, extrapyramidal features and sensory disturbance with small fiber neuropathy, are now recognized as part of the disease [ 2 , 3 ].…”