Abstract

ALS: THE BASICS AND BEYOND

Paras Ravishankar Shukla, Harsh Chintamani Jadhav, Atharva Gajanan Puri, Pankaj Subhash Gavit, Prajwal Vinayak Padekar, Madhuri Vijay Supekar

Abstract

Lou Gehrig's disease, sometimes also known as amyotrophic lateral sclerosis (ALS), is an incurable neurological disorder affects motor neurones in the brain and spinal cord. Primarily recognised by Jean-Martin Charcot in 1869, ALS develops muscle weakness, paralysis, and finally respiratory failure. Still completely unknown is the aetiology; environmental and genetic components have been connected. Pathologically, ALS's accumulation of proteins and motor neurone destruction demonstrate Pathophysiologically, it correlates with the development of oxidative stress, mitochondrial dysfunction, and neuroinflammation. Treatment is basically supportive; riluzole and edaravone are FDA-approved medications with moderate advantages; confirmation is dependent on clinical signs as well as symptoms, electromyography, and exclusion of other illnesses. Multidisciplinary cooperation with a value on inflammation of the neurones, stem cell treatments, and gene therapy has founded present investigations (2022–2024) on controlling symptoms. Especially remarkable advancements include ongoing investigation of antisense oligonucleotide therapies for C9orf72-related ALS and the Food and Drug Administration's approval of tofersen for SOD1-ALS in 2023. Under investigations are pharmacological treatments using small compounds focussing at mitochondrial function and protein aggregation. Future treatments might call for combination medications covering several pathways in addition to customised approaches depending on genetic profiles. About 2–3 per 100,000 people worldwide suffer with ALS. Well-known people impacted are Lou Gehrig and Stephen Hawking. Research advances bring cautious hope for better therapies and maybe a cure for this terrible illness. Early symptoms of ALS include trembling of the muscles and lack of energy, growing to paralysis and difficulties breathing, whereas mental impairment may occur in later stages and progression varies; most people remain 2-5 years after symptom onset.

Keywords: Motor neuron disease, Lou Gehrig's disease, Muscle weakness, Progressive paralysis, Stem cell research.