![]() Diagnosis combines clinical examination, nerve conduction studies and electromyography, and laboratory tests, and is typically made using the revised El Escorial criteria (Table 2). 7 This, in turn, delays initiation of therapies that may improve symptoms and slow disease progression. The disease has a heterogenous presentation and is a diagnosis of exclusion, which can lead to diagnostic delays of up to 1 year (Table 1). Since the first ALS genetic mutation was identified in 1993, more than 120 are now known to contribute to the disease.4 Several other potential risk factors have been investigated, including exposure to pesticides, chemicals, and heavy metals military service athleticism smoking and viruses. The greatest risk factors for ALS are age and family history, with numerous genetic aberrations now identified. ![]() The overall prevalence in the United States is approximately 5 per 100,000 individuals. 4 More than 5000 patients in the United States receive a diagnosis of ALS each year, and an estimated 20,000 are living with the disease. ![]() Sporadic ALS is diagnosed at an average age of 55 years, but the familial form of the disease may manifest much earlier. In the United States, men are almost twice as likely as women to develop the sporadic form. In both familial and sporadic forms of ALS, the symptoms are the same. The majority of patients are diagnosed with sporadic ALS, whereas about 10% are diagnosed with the familial form of the disease. There is no cure and just 2 drugs are approved for the disease, each of which can slow progression somewhat in selected patients. 3 Most patients die of respiratory failure within 2 to 5 years of onset. 1,2 A strong association between frontotemporal dementia (FTD) and ALS is now recognized, with between 20% and 50% of those with the disease developing FTD. This article highlights the oral tyrosine kinase inhibitor masitinib the antisense drug tofersen the humanized monoclonal antibody C5 complement inhibitor ravulizumab-cwvz and mesenchymal stem cell (MSC)-neurotrophic factor (NTF) cells, a proprietary platform that induces autologous bone marrow-derived MSCs to secrete high levels of NTFs.Īmyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder characterized by the loss of cortical and spinal motor neurons, leading to weakness, muscle atrophy, and, in a substantial number of patients, cognitive impairment. Recent developments in the understanding of the underlying pathophysiologic processes that contribute to ALS have led to the development of numerous investigational therapies, with several now in phase 3 trials. Two drugs, riluzole and edaravone, are currently FDA approved for the treatment of ALS, and each provides modest benefits in mortality and/or function. The economic cost of the disease is not clear, with estimates ranging from about $64,000 per year to $200,000. The disease stems from death of upper and lower motor neurons leading to degeneration of motor pathways and the paralytic effects of the disease. Most patients die within 2 to 5 years of diagnosis. Amyotrophic lateral sclerosis (ALS) is a devastating, fatal neuromuscular disease.
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