News

A new ALS Association partnership with the ALS Investment Fund II seeks to raise $100 million in new funding to advance amyotrophic lateral sclerosis (ALS) therapy development. This second venture fund follows one that originated in the Netherlands and raised $25 million in 2.5 years to support…

With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough Summit. From diagnosis and clinical trial design to manufacturing, pricing strategies, and ethical concerns, gene therapy — both its high…

Treatment with oral reldesemtiv appears to slow the deterioration of function — including breathing capacity and muscle strength — in amyotrophic lateral sclerosis (ALS) patients, regardless of whether or not they were taking additional medications such as Radicava (edaravone) or Rilutek (riluzole), a new…

With a $100,000 grant, the FTD Disorders Registry has received a boost from CurePSP, a nonprofit organization working to enhance awareness, education and care for prime-of-life neurodegenerative diseases. Launched in 2017 by the Association for Frontotemporal Degeneration (AFTD) and the Bluefield Project to Cure FTD — with…

A new study identified a group of sporadic amyotrophic lateral sclerosis (ALS) patients whose disease may arise from a group of “jumping genes” unleashed when TDP-43 protein accumulates in clumps in the brain and spinal cord. These “jumping genes” — which can randomly hop from one location on…

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.

When intrastate rivals University of Washington (UW) and Washington State University (WSU) met Nov. 29 for the storied Boeing Apple Cup Series, the battle wasn’t just about football — it was also about amyotrophic lateral sclerosis (ALS). The teams’ fan bases were vying to see which…

Cromolyn sodium — a U.S. Food and Drug Administration (FDA)-approved anti-inflammatory medication — delayed disease onset and prevented motor neuron degeneration and loss in a mouse model of amyotrophic lateral sclerosis (ALS), researchers report. These neuroprotective effects were accompanied by a decrease in inflammation, highlighting the potential…

Mitsubishi Tanabe Pharma America (MTPA) has enrolled a first patient in a Phase 3 clinical trial of an oral suspension formulation of edaravone in treating amyotrophic lateral sclerosis (ALS). An intravenous form of edaravone (given directly into the bloodstream), developed by Mitsubishi Tanabe Pharma and sold under the brand…

A dissolving oral film form of riluzole, called Exservan and able to be taken by amyotrophic lateral sclerosis (ALS) patients without a need for water, has been approved by the U.S. Food and Drug Administration (FDA). Riluzole was the first ALS treatment, approved by the FDA over two decades…