Decreasing mobility is a challenge faced by every person living with ALS at some point in their disease journey. Finding ways to help people maintain their independence and prevent potential harms caused by everyday living activities is a priority for the ALS community.
The ALS community has the opportunity to encourage The Food and Drug Administration (FDA) to approve Amylyx Pharmaceutical’s new drug application for AMX0035.
An experimental treatment given to a marathon runner in the early stages of ALS led to a significant and long-lasting reduction in levels of toxic dipeptide repeat proteins in the cerebrospinal fluid. The treatment, an antisense oligonucleotide directed at suppressing a repeat expansion in the non-coding region of chromosome 9 open reading frame 72, or C9ORF72, has not been proven to show a clinical benefit yet, but the patient's condition has remained stable since the infusion therapy protocol was started in 2019.
A small business owner and artist first started selling his work to pay the bills. Now, he's spreading awareness about ALS in memory of his father who died in 2017.
Scientists from two independent research teams have discovered how the mislocalization of a protein, known as TDP-43, alters the genetic instructions for UNC13A, providing a possible therapeutic target that could also have implications in treating amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), and other forms of dementia. ALS and FTD are two neurodegenerative disorders in which many cases are linked by mislocalization of TDP-43, where instead of being primarily located in the nucleus of the cell where genes are activated, it forms aggregates outside the nucleus in multiple neurodegenerative diseases. Rare mutations in the TDP-43 gene are known to cause ALS, but almost all cases of ALS show mislocalization of TDP-43. The studies were published in Nature.
Serum asymmetric dimethylarginine (ADMA) level is an independent biomarker of disease progression and prognosis in amyotrophic lateral sclerosis (ALS), according to a study published in the European Journal of Neurology.