Repeated muscle injections with Engensis (VM202), Helixmith’s investigational non-viral gene therapy, were generally safe and well-tolerated in people with amyotrophic lateral sclerosis (ALS), according to top-line data from a Phase 2a clinical trial. While the sample size was too small to determine the therapy’s efficacy, muscle biopsies were…
Engensis Gene Therapy Found Safe in Small Phase 2a Trial
Is it just me, or are people speaking at a faster clip nowadays? Yeah, maybe it’s just me. It seems I’ve become hypersensitive to the various speeds and rhythms we use to communicate with each other, especially now that I live with dysarthria, a symptom of ALS that affects…
Higher blood levels of antibodies against the viral protein HERV-K ENV are associated with a better survival rate and improved outcomes in people with amyotrophic lateral sclerosis (ALS), a new study shows. These findings further support the therapeutic potential of GNK301, an experimental antibody against HERV-K ENV being…
To help expand research into the treatment and cure of amyotrophic lateral sclerosis (ALS), the Blazeman Foundation for ALS has donated $800,000 to the University of Maryland School of Medicine (UMSOM). The funds will be used to boost research at the University of Maryland Brain and Tissue Bank,…
Power wheelchairs are expensive, and while Medicare covers the base cost, many beneficial features are an additional out-of-pocket expense for those already facing the high cost of living with ALS in the United States. Nine years ago, we began the process of ordering a power wheelchair for…
Treating patients with their own immune regulatory T-cells (Tregs), in combination with IL-2 — a protein that boosts the cells’ immunosuppressive function and survival — is generally safe and shows potential to slow disease progression in people with amyotrophic lateral sclerosis (ALS). That’s according to a small Phase…
The cerebrospinal fluid of people with sporadic amyotrophic lateral sclerosis (sALS), but not of those with familial forms of the disease, promoted ALS-specific molecular, cellular, and motor abnormalities in healthy mice, a study showed. Apolipoprotein B-100 (apoB100), a protein involved in cholesterol transport, was identified as the main…
I’ve been living with ALS for 12 years now, and it’s certainly taught me many lessons. They’ve involved having patience and perseverance, as well as getting back up over and over. The most important lesson, however, has been to let the present moment be what it is, instead of…
Altered forms of the SOD1 protein are found in the spinal cord nerve cells of people with all types of amyotrophic lateral sclerosis (ALS), not just those with mutations in the gene providing instructions for making the protein, a study reported. “The results suggest this abnormal protein contributes to cell…
The European Medicines Agency (EMA) has agreed to review an application seeking the conditional approval of AB Science’s masitinib, now Alsitek, for the treatment of amyotrophic lateral sclerosis (ALS). Conditional approval is granted to a medication whose immediate availability fulfills an unmet medical need, when its preliminary benefits are…
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