News

Mutations in the FUS gene, one of the most common causes of familial amyotrophic lateral sclerosis (ALS), increase the number of branches extending from the axons of motor nerve cells, so that no clear path exists for these neuronal “arms” to transmit information — in the form of electrical impulses —…

People with amyotrophic lateral sclerosis (ALS) have higher blood serum levels of neurofilament light chain (sNfL) than do healthy people, a new study found. Its work also further supported sNfL’s usefulness as a marker of likely disease progression and outcomes, with higher concentrations indicating poorer survival. The research, “…

Measuring lung function can help to predict overall disease progression in people with amyotrophic lateral sclerosis (ALS), a study suggests. “Classifying Amyotrophic Lateral Sclerosis Patients by Changes in Forced Vital Capacity: A Group-Based Trajectory Analysis” was published in the American Journal of Respiratory and Critical Care…

Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest.    Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…

Excessive energy demands to overcome gravity in nerve cells directed downward — such as those involved in the control of limb movements — may explain why amyotrophic lateral sclerosis (ALS) affects certain motor neurons while leaving others untouched, a study has proposed. As people age, nerve fibers disposed vertically…

Disarm Therapeutics has announced the publication of two studies that further researchers’ understanding of the structure and function of SARM1, a key protein in cellular degeneration. The new data may aid in the design of therapies for multiple diseases, including amyotrophic lateral sclerosis (ALS). The findings were published in…

Orphazyme announced that it and Worldwide Clinical Trials will continue their collaboration through the long-term extension of a Phase 3 study into arimoclomol, a potential oral treatment for amyotrophic lateral sclerosis (ALS). ORARIALS-01 is a randomized, placebo-controlled and double-blind trial (NCT03491462) underway at 30 centers across North America…

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

Amyotrophic lateral sclerosis (ALS) progression is associated with higher levels of specific lipids in the spinal cord and changes in their metabolism, a study in a rat model of the disease reports. These findings may represent a defense mechanism against oxidative damage, as well as a potential treatment route for…

A newly developed robotic neck brace may improve quality of life for people with amyotrophic lateral sclerosis (ALS) and allow researchers a more detailed assessment of head and neck movements, as well as disease progression, for people with this condition. The brace was described in a pilot study, titled…