Note: This story were updated Feb. 14, 2023, to correct that ICER analyses found both Radicava ORS and Relyvrio are not cost-effective for the treatment of ALS. The recently approved therapies Relyvrio (sodium phenylbutyrate and taurursodiol) and …
The exact cause of amyotrophic lateral sclerosis (ALS) is largely unknown, but a number of factors including genetics, environmental exposures, and lifestyle habits have been linked to an increased risk of ALS.
In the Jan. 23 episode of “The Happiness Lab” podcast, Simone Stolzoff, author of the book “The Good Enough Job: Reclaiming Life from Work,” argues that we should stop hunting for the perfect job. Stolzoff and podcast host Laurie Santos, a psychology professor, discuss how society’s view of work…
Cytokinetics has awarded $20,000 grants to five patient advocacy organizations, including two serving the amyotrophic lateral sclerosis (ALS) community, under its fifth annual Communications Grant Program. Each grant is intended to support the nonprofit’s outreach, engagement, and awareness efforts, allowing the organizations to broaden their work in underserved…
A new pig model of amyotrophic lateral sclerosis (ALS) more accurately reflects the gradual motor neuron loss and disease progression seen in patients, and it may speed the development of ALS treatments, included gene and cell-based therapies, researchers report. The model carries a mutation in the SOD1 gene, an…
Utreloxastat (PTC857), an investigational therapy for people with amyotrophic lateral sclerosis (ALS), was safe, well tolerated, and displayed promising pharmacological properties in healthy people, according to a first-in-human Phase 1 study. Based on these findings, the therapy’s developer, PTC Therapeutics, has launched a Phase 2 trial (NCT05349721)…
I’ve read dozens, if not more, of the kind of column I’m about to write: first-person narratives that offer advice to one’s younger self, full of lessons learned from difficult life experiences. Before my late husband, Jeff, was diagnosed with ALS, I devoured these columns, always looking for some…
Elevated levels of high-density lipoprotein (HDL) — commonly called the “good” cholesterol — are significantly associated with a poorer survival rate among people with amyotrophic lateral sclerosis (ALS), according to a population-based study in the Netherlands. In contrast, levels of total cholesterol and low-density lipoprotein…
Now that my husband, Todd, is paralyzed and uses a noninvasive ventilator, leaving the house is too hard on him. So when we have a date night, we watch a movie at home. Last weekend, we watched “Good Night Oppy,” a documentary about twin rovers that NASA…
The U.S. Food and Drug Administration (FDA) has announced an advisory committee meeting seeking advice on whether to approve Biogen‘s investigational therapy tofersen for the treatment of amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations. The public meeting, to be held online March 22, by…
