ET-101, Eikonoklastes Therapeutics’ experimental gene therapy for amyotrophic lateral sclerosis (ALS), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). Orphan drug status is given to investigational therapies with the potential to significantly benefit people with life-threatening or chronically debilitating diseases that affect…
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The Lifestyle Healing Institute (LHI), which uses a complementary and alternative medicine approach to care, is touting the success of an amyotrophic lateral sclerosis (ALS) patient whose mobility and quality of life are said to have improved following treatment. The case of Tod, a 60-year-old patient, demonstrates that…
The U.S. Food and Drug Administration (FDA) notified Brainstorm Cell Therapeutics that it will not accept for review a company application asking that its cell-based therapy NurOwn be approved as a treatment for amyotrophic lateral sclerosis (ALS). The FDA decision, in the form of a refusal to file…
Radicava Oral Suspension, an oral formulation of edaravone, has been approved in Canada for treating amyotrophic lateral sclerosis (ALS). Mitsubishi Tanabe Pharma Canada (MTP-CA), the subsidiary of Mitsubishi Tanabe Pharma America that markets Radicava in Canada, has announced plans to make the oral formula available to patients…
The investigational cell-based therapy NurOwn may slow disease progression in people with amyotrophic lateral sclerosis (ALS) who have less severe disease, according to analyses from a Phase 3 trial. Researchers’ analyses excluded patients with the lowest scores on the ALS Functional Rating Scale–Revised (ALSFRS-R) or in individual…
Early declines in neurofilament light chain (NfL), a biomarker of nerve cell damage, after treatment with tofersen may predict a slower disease progression over time in people with SOD1-associated amyotrophic lateral sclerosis (ALS). Patients who received tofersen early also experienced a significantly slower worsening of clinical function than…
Continuous monitoring of certain biomarkers in people with amyotrophic lateral sclerosis (ALS) may inform about the risk of disease progression and the response to edaravone, according to interim data from the ongoing REFINE-ALS biomarker study. Mitsubishi Tanabe Pharma America (MTPA), the developer of edaravone, recently shared these findings…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Stealth Biotherapeutics’ investigational therapy SBT-272 for the treatment of amyotrophic lateral sclerosis (ALS). Aimed at speeding the development of new treatments for rare, life-threatening diseases, orphan drug status offers regulatory support and certain financial incentives…
Four years after its discovery via an artificial intelligence (AI) platform, Verge Genomics will test its amyotrophic lateral sclerosis (ALS) treatment candidate VRG50635 — a PIKfyve inhibitor — in a first-in-human trial. The Phase 1 clinical trial (ISRCTN14792372), which is now enrolling participants, is being conducted at the…
CuATSM, a potential amyotrophic lateral sclerosis (ALS) therapy that’s now in clinical trials, may work by altering how certain cells in the brain generate energy, a new study suggests. Researchers say these findings may help to identify patients most likely to respond to the experimental treatment. “Screening patient derived…
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