RT001, an investigational treatment for amyotrophic lateral sclerosis (ALS), shows promising signs that it can slow disease progression, particularly among patients with more severe disease, according to data from a completed Phase 2 pilot trial. While these findings indicate improvements in patients on RT001 treatment, statistical significance was…
News
Amylyx Pharmaceuticals has donated data from the CENTAUR Phase 2 trial to the PRO-ACT database — the largest collection ever compiled of de-identified amyotrophic lateral sclerosis (ALS) patient records from clinical trials. CENTAUR (NCT03127514) evaluated the safety and efficacy of Amylyx’s investigational treatment AMX0035 against a placebo…
Kulicke & Soffa Industries has announced a multi-year donation that ultimately will increase amyotrophic lateral sclerosis (ALS) patients’ access to potentially life-saving treatments. The contribution, for which an amount was not specified, will establish and support the multicenter expanded access protocol (EAP) companion program to the HEALEY ALS…
AMX0035 — Amylyx Pharmaceuticals’ experimental combination therapy for amyotrophic lateral sclerosis (ALS) now under review for approval in the U.S. — alters the activity of genes involved in several disease-relevant pathways, according to a study in lab-grown cells. Notably, the two-compound oral treatment was seen to change the activity of many…
The levels of five small RNA molecules, called microRNAs, inside tiny vesicles in the blood of people with amyotrophic lateral sclerosis (ALS) differ significantly from those of people without ALS and may offer a way of accurately and quickly diagnosing the disease, a study suggests. Importantly, its researchers evaluated…
A treatment known as VPA-Li, a combination of lithium carbonate and valproic acid, may slow disease progression and improve quality of life for people with amyotrophic lateral sclerosis (ALS), according to data from a small Phase 2 clinical trial. Favorable trends in survival and lung health were also observed…
Stem cells engineered to mature into nerve-supporting cells that produce GDNF — a key factor for motor neuron survival — were safely delivered into the spinal cord of people with amyotrophic lateral sclerosis (ALS), according to data from a Phase 1/2a clinical trial. These cells, dubbed CNS10-NPC-GDNF, were…
In a reversal, an advisory committee of the U.S. Food and Drug Administration (FDA) now says that the current evidence of efficacy of Amylyx Pharmaceuticals’ AMX0035 is sufficient to support its approval for amyotrophic lateral sclerosis (ALS). The committee voted 7–2 in favor of AMX0035, Amylyx announced…
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…
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…
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