The rarity of amyotrophic lateral sclerosis (ALS) in children makes it impossible to realistically conduct clinical trials of experimental medicines in this population, researchers across Europe argue in a paper. “Given the absence of consensus on pediatric ALS as a disease entity, the extremely low prevalence of ALS in…
Clinical Trials in Pediatric ALS ‘Simply Not Feasible,’ Scientists Argue
“What’s the buzz? Tell me what’s a-happening, Why should you want to know? Don’t you mind about the future? Don’t you try to think ahead? Save tomorrow for tomorrow, Think about today instead.” — Andrew Lloyd Webber and Tim Rice, “What’s the Buzz,” from the rock opera “Jesus Christ…
After a two-year hiatus, the Muscular Dystrophy Association (MDA) is bringing back its in-person Wings Over Wall Street fundraiser gala in New York City, with proceeds going to support MDA research into promising amyotrophic lateral sclerosis (ALS) treatments. The June 9 event, starting at 6 p.m. EST at…
I use many strategies to keep myself engaged in life and avoid getting pulled down mentally by my ALS. Being a “go-along” is one of those strategies, and it’s a perfect partner to showing up. But being a go-along — that is, accompanying my husband on his various errands…
The Barrow Neurological Institute in Phoenix, Arizona, is calling on people with amyotrophic lateral sclerosis (ALS) and their family members and friends to participate in a biomarker study that aims to create a bank of biological samples and clinical data collected over time. Data will be made available…
One-time delivery of a CRISPR-based gene therapy significantly reduced the buildup of toxic RNA molecules resulting from C9ORF72 mutations in cells and mouse models of amyotrophic lateral sclerosis (ALS), the therapy’s developer, Locanabio, reported. The approach was designed using the company’s CORRECTx platform, which uses CRISPR technology to…
After several years of support, the late-stage biopharmaceutical company Cytokinetics is reaffirming its commitment to the ALS Association and its efforts to battle amyotrophic lateral sclerosis (ALS) and assist the ALS community. In addition to backing nationwide organizational efforts, Cytokinetics has, for more than a decade, provided support…
A couple weeks ago, when my husband, Todd, told me he wasn’t going to attend our kids’ end-of-the-year events at school — including our son’s band concert and our daughter’s choir concert and dance recital — it was a reality check for me about where we are in this ALS…
The estimated 2017 U.S. prevalence of individuals with amyotrophic lateral sclerosis (ALS) was 24,821, or 7.7 per 100,000 people, according to a recent report from the National ALS Registry. While a statistical model was used to account for potentially missing data, the research team emphasized that this number is…
Treatment with AMX0035 significantly prolonged the time that people with amyotrophic lateral sclerosis (ALS) in the CENTAUR clinical trial did not require permanent ventilation or tracheostomy by over seven months, a new study shows. “People living with ALS often require medical interventions like mechanical ventilation as the disease…
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