AveXis and REGENXBIO Sign Licensing Agreement to Develop Therapies for ALS and Rett syndrome

AveXis and REGENXBIO have signed a new licensing agreement to develop therapies for a particular form of ALS and for Rett syndrome. They already have an agreement that covers spinal muscular atrophy therapies. The form of amyotrophic lateral sclerosis that the new agreement covers is the one caused by mutations…

“How about we try that new place that just opened up near the mall?”  I knew my husband was referring to the new restaurant in town. In the old days, I’d simply agree and that would be that. But now, living with ALS, I’ve learned a thing or two about…

ALS patients compensate for neuron loss in some brain regions by stepping up activity in other regions, according to a study. The finding suggests that analyzing patients’ brain networks could help doctors determine the state of their disease and monitor its progression. The study, “Functional reorganization during cognitive function…

People dying of terminal diseases like amyotrophic lateral sclerosis (ALS) and inmates on death row have something in common: their outlooks are more positive than might be expected. A recent study examining attitudes on impending death, “Dying is Unexpectedly Positive,” appeared in the journal Psychological Science. Death is an important part…

Brad Gildenblatt was diagnosed with amyotrophic lateral sclerosis in 2014. Three years later, his ALS had progressed to the point where he was unable to travel from Dallas to Oklahoma to see his only child, Caitlin, graduate from Oklahoma State University. However, when one of the deans of the university found out,…

Flex Pharma, which develops therapies to reduce the cramps and shaking that are hallmarks of ALS and other neurological diseases, has a new CEO. Dr. William McVicar, head of the company’s research and development arm, will assume the interim chief executive’s post on July 3. He replaces Dr. Christoph Westphal, who…

The Muscular Dystrophy Association, in collaboration with the Lewin Group, has calculated the average cost of caring for three of the most common neuromuscular disorders. MORE: Most with chronic conditions find the Affordable Care Act beneficial The study looks at both medical and non-medical costs associated…

Having a service dog can greatly enhance the life of someone living with a chronic disease. They allow patients to regain some of their independence by helping with small everyday tasks like opening and closing doors, fetching meds, acting as a prop or support as their owner stands, switching on lights and attracting attention…

American Airlines has raised more than $800,000 at its second annual American Airlines Charity Golf Tournament to support the research initiative Answer ALS. The tournament and a fund-raising auction took place in American’s home base of Dallas, May 24 and 25. “We are proud to partner with Answer ALS…

Insilico Medicine has launched ALS.AI, a drug discovery platform for new amyotrophic lateral sclerosis (ALS) treatments. It uses a powerful new analytical approach involving “deep learning” to seek out new therapies for the disease — and to evaluate whether existing drugs used to treat other diseases can be repurposed for treating ALS.