Rare Disease Groups Welcome FDA’s Embrace of ‘Real World’ Data in Clinical Trials

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…

The Montreal Neurological Institute of McGill University and Thermo Fisher Scientific have recently partnered to accelerate the understanding of neurological disease by focusing on about 30 proteins associated with amyotrophic lateral sclerosis (ALS) and other brain diseases. The collaboration is part of…

At a time of unprecedented polarization in Congress, two U.S. lawmakers — one Republican, one Democrat — are stressing the urgency of working across the aisle to help the estimated 30 million Americans with rare diseases. Rep. Leonard Lance (R-New Jersey) and Sen. Amy Klobuchar (D-Minnesota) spoke to more…

One of the most frustrating symptoms of living with a chronic illness is brain fog. There are medications to treat many symptoms of chronic diseases, but sadly there isn’t yet a pill that takes away brain fog. However, there are ways to deal with it so patients can minimize its effects and…

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…