The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December, with the aim of empowering leaders to start non-profit organizations and research programs to help people with rare diseases. “The RareLaunch program is…
“Your lifelong membership is free, Keep a-giving each brother all you can. Oh, aren’t you proud to be in that fraternity, The great big brotherhood of man?” Those lines, written by Frank Loesser for the musical…
Novus Therapeutics has enrolled the first patient in its Phase 2a trial of AT-1501 in adults with amyotrophic lateral sclerosis (ALS), the company announced in a press release. The trial is enrolling up to 54 adults with ALS across up to 12 U.S. treatment sites. Currently,…
Although it’s hard to find much to celebrate in 2020, this year happens to mark an important milestone for my husband and me. I’m celebrating achieving 10 years of living with ALS, and for my husband, 2020 marks a decade of his role as a caregiver — to me. I…
After working for months behind the scenes, the ALS Association is calling a strengthened Accelerating Access to Critical Therapies for ALS Act (ACT) a key step toward swifter development of new therapies. The revised bill (HR 8662/S. 4867) calls for $100 million over five years for amyotrophic…
A newly identified type of immune cell — a kind of immature neurophil or white blood cell — was found to be responsible for the regeneration and survival of eye and spinal cord nerve fibers that is known to be possible in mice with induced nerve injury,…
A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover of an upcoming calendar in the “Same But Different” contest to raise awareness about rare disorders. “A Lovely Day Out in Kew Gardens,”…
Eli Lilly and Company has acquired Disarm Therapeutics and its emerging therapies designed to prevent axonal degeneration in neurodegenerative disorders, including amyotrophic lateral sclerosis (ALS), multiple sclerosis, and peripheral neuropathy. Disarm’s new class of disease-modifying treatments, called SARM1 inhibitors, target the central driver of axonal…
Last week, we had an early snowfall, even for Michigan’s Keweenaw Peninsula. Snow frosted the tips of trees and blanketed the grass, leaving small bits of green peeking through. I posted a picture on social media and wrote, “It’s beginning to look a lot like Christmas. Gearing up for…
Bristol Myers Squibb has entered a five-year research collaboration with the machine learning startup insitro to discover and develop new therapies for amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Machine learning is a form of artificial intelligence that uses algorithms to analyze large amounts of data, learn…
