In his 10 months on the job, Commissioner Scott Gottlieb of the U.S. Food and Drug Administration is earning praise for his efforts to make clinical trials for new therapies more flexible and responsive to the needs of rare disease patients. From cystic fibrosis to epidermolysis bullosa, the FDA…

Researchers have identified genes that promote amyotrophic lateral sclerosis (ALS), using the gene-editing technology CRISPR-Cas9. The findings represent not only another piece of information to understand the molecular mechanisms triggering ALS, but also support the newly identified genes as potential targets for future therapeutics. The study “CRISPR–Cas9 screens in human cells…

Researchers have made advances in understanding how angiogenin, a key protein associated with amyotrophic lateral sclerosis (ALS), enters and is transported inside nerve cells. Although the mechanisms behind those events were found to be more complex than previously thought, the findings may help to find new targets and new therapies…

https://vimeo.com/165626427 There are currently around 30,000 people in the U.S. living with ALS and that number grows by approximately 15 each day. MORE: Learn about Nanci, a much-loved Hollywood publicist who was diagnosed with amyotrophic lateral sclerosis in 2014 This ALS Association of Texas video explains amyotrophic lateral…

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that attacks motor neurons, leading to wastage of the muscles. This affects every area of the body and as the disease progresses, patients begin to experience speech impairments and will eventually lose the ability to speak. MORE: Explaining the progression of ALS…

Nerve cells that control movement change their size as ALS progresses, becoming larger than normal at first but then ending up much smaller, a mouse study showed. An important finding was that the changes can prevent nerve cells from facilitating communication between the brain and muscles. The research provided scientists with…

Genervon Biopharmaceuticals says its efforts to develop treatments for ALS and other neurodegenerative diseases continue to focus on a factor that regulates stem cells’ evolution into nerve cells. It continues working on Phase 3 clinical trial plans, it added. The company outlined its treatment-development strategy at the…

Scientists have gained greater insight into the structure of the nucleolus, a cellular organelle that has been implicated in diseases such as amyotrophic lateral sclerosis (ALS). By honing in on the molecular events surrounding ALS, scientists can start to understand how the disease develops, which could lead to new…

The more a person is exposed to diesel exhaust, the greater the chance they will develop ALS, a Harvard University study on Denmark reports. The team will present the findings at the American Academy of Neurology‘s 70th Annual Meeting in Los Angeles, April 21 to 27. The presentation is titled “…

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease characterized by the deterioration of muscles in the body, leading to loss of movement, an inability to speak and swallow, and eventually, breathe. According to medscape.com, the rate at which muscle deterioration occurs will differ from patient to…