Tofersen, an experimental treatment for amyotrophic lateral sclerosis (ALS) patients with mutations in the SOD1 gene, failed to significantly slow the rate of disease progression relative to a placebo in a Phase 3 trial. These top-line findings mean that the VALOR study (NCT02623699), which is testing tofersen in…
Like most folks who live with ALS, I tend to get stuck in the shortsighted day-to-day management of my symptoms. Over the years, I’ve developed several strategies that help me change my mental focus from a short view to a longer one in which I’m open to the possibilities of…
The ALS Association is reminding amyotrophic lateral sclerosis (ALS) patients and caregivers that the open enrollment period for Medicare recipients to make changes to their existing coverage runs through Dec. 7. The open enrollment period for altering coverage opened Friday. After Dec. 7. changes will be allowed only after…
Note: This story was updated Oct. 19, 2021, to note that the trial is taking place at 90 clinical sites and to update the contact information, based on additional information from the company. A Phase 3b clinical trial is recruiting adults with amyotrophic lateral sclerosis (ALS) to assess the…
Amgen and Neumora Therapeutics announced that they are collaborating to develop precision therapies for amyotrophic lateral sclerosis (ALS) and other brain diseases. The partnership will leverage each of the company’s expertise, with the goal of bringing to the market effective therapies targeted at those patients most likely…
My husband, Todd, began monitoring his breathing after he was diagnosed with ALS. We were told that he’d need to have surgery to insert a feeding tube before his forced vital capacity (FVC) dipped below 50%, and so he had it measured every few months at an ALS clinic.
A signaling protein called neurturin promotes the development of muscles with greater endurance, leading to better exercise performance and coordination, according to a new mouse study. The results suggest that therapeutic approaches using neurturin — for example, using a gene therapy that encodes the protein — might be useful…
“And so castles made of sand Fall into the sea eventually.” — Jimi Hendrix From time to time, given my predilection for quoting songs in my columns, I’m asked whether a song triggers content, or if it’s subject matter in search of lyrics. The answer is, circumstantially, either. On…
The Rare Disease Diversity Coalition (RDDC) awarded $600,000 in grants to ease the disparities faced by rare disease patients of color. These Impact Rare Disease Solution grants will go five RDDC steering committee working groups, which aim to identify problems for rare disease communities and advocate for solutions. The five…
An investigational treatment for amyotrophic lateral sclerosis (ALS), SBT-272 was found to sustainably reach different brain regions, and to protect mitochondria — a cell’s energy source — from TDP-43 toxic aggregates in a mouse model of the disease. “We are excited about the promise of SBT-272 as a potential therapeutic…
Get regular updates to your inbox.