It’s been a decade since the first UMass ALS Cellucci Fund team first participated in the Boston Marathon to raise funds to support University of Massachusetts Medical School (UMMS) researchers in their quest to find better treatments for amyotrophic lateral sclerosis (ALS). So, this year is significant. The…
Help has come and gone over the past decade since my husband, Todd, has had ALS. When it became too difficult to travel four hours away to an ALS clinic, Todd went to a local neurologist who managed his care. After a couple years, his neurologist left the area, and…
Seelos Therapeutics has announced plans to start patient dosing in a pivotal Phase 2b/3 trial testing its experimental therapy SLS-005 (trehalose), designed to prevent protein clumping in cells, in adults with amyotrophic lateral sclerosis (ALS) by the close of September. The study is part of the HEALEY ALS Platform…
Rare mutations in the TP73 gene, which is involved in the regulation of a cell’s life cycle, may put a person at greater risk of amyotrophic lateral sclerosis (ALS), according to a genetic analysis of nearly 2,900 sporadic ALS patients. Such mutations were found to affect the maturation and survival of…
Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…
Diminished activity in the C9orf72 gene that is linked with amyotrophic lateral sclerosis (ALS) affected the neuromuscular junction — where nerve and muscle cells connect — in a zebrafish model, establishing the model as a disease research tool and the gene’s role in ALS symptoms. Zebrafish in this C9orf72 loss-of-function model…
Men who played professional soccer were nearly four times more likely to develop a neurodegenerative disorder, including a motor neuron disease such as amyotrophic lateral sclerosis (ALS), than men of similar age in the general population, a long-term analysis found. Risk was greatest in players with longer careers and…
The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ketamine, PharmaTher’s investigational therapy for amyotrophic lateral sclerosis (ALS). Orphan drug status is intended to encourage the development of therapies for rare diseases affecting fewer than 200,000 people in the U.S. It provides…
Driving in our small town is usually pretty chill. People let each other in and don’t cut one another off. But right now, the roads are busier than normal. It’s construction season, and we have many out-of-town tourists. People come from big cities, where they live with more stress and…
Actemra (tocilizumab), an approved treatment for rheumatoid arthritis, was found to be safe and well-tolerated, and to reduce a key marker of inflammation in amyotrophic lateral sclerosis (ALS) patients with evidence of systemic inflammation in a Phase 2 study. Trial results did not demonstrate that Actemra could slow ALS disability…
Get regular updates to your inbox.