Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

After my husband, Todd, was diagnosed with ALS, we thought he would have at most five years to live. We started with only two major goals: take a family vacation together with our toddler and preschooler, and build an accessible home. We did both of those things within the…

Note: This story was updated April 6, 2021, to note that the study is expected to conclude late this summer, instead of 2024. The ALS Association, Muscular Dystrophy Association (MDA), and ALS Finding a Cure have given an additional $1.1 million to support work on a cell therapy…

“They’re gonna put me in the movies, They’re gonna make a big star out of me, … And all I gotta do is act naturally.” — Johnny Russell Two weeks ago, I referenced several catchphrases from…

Biogen will not offer early access to tofersen, its experimental therapy for familial amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene, at least until data from its pivotal clinical trial are analyzed. Now in its Phase 3 portion, the study (NCT02623699) is testing the…

Retrotope has dosed the first patients in its recently-initiated Phase 2 clinical trial evaluating RT001 for the treatment of amyotrophic lateral sclerosis (ALS). Enrollment is open across four European sites for patients ages 20–75 whose disease symptoms began less than three years ago and do not need…

Roche Canada’s Artificial Intelligence Centre of Excellence (AI CoE), in collaboration with Answer ALS and EverythingALS, opened a challenge that asks the global artificial intelligence (AI) community to model solutions for key questions in amyotrophic lateral sclerosis (ALS). Called End ALS Challenge, the digital competition also…

Note: This story was updated March 31, 2021, to note that the ALSummit trial has been approved by the U.S. Food and Drug Administration but will proceed only in Korea; there are no plans to open additional sites in the U.S. or elsewhere. A Phase 3 clinical trial is…

“What the heck happened to that guy?” a young boy called loudly to a woman leading him and other preschool children across the parking lot. I was loading my husband, Todd, into the back of our accessible van, having just gotten our COVID-19 vaccinations. It was the first time…

A new feature of Cox’s accessible web remote enables people with amyotrophic lateral sclerosis (ALS) and others with disabilities to control their TVs using their eyes. The free remote also can be used with other assistive technologies, such as sip-and-puff. “We are committed to improving our products to…