Mutations in FUS Gene Affects Growth and Workings of Motor Neuron Axons, Study Shows

During my early childhood, I often played Chutes and Ladders. For those who are unfamiliar with the board game or have a foggy memory, the board is made up of 100 numbered squares. Beginning in the lower-left corner, players move a certain number of spaces, determined by…

What does smiling have to do with ALS? Or happiness, contentment, or feeling positive? These expressions of our sense of well-being are important to those of us living with ALS. According to a study of 224 ALS patients, participants who had good emotional well-being experienced a slower progression…

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,…

“So tell us your story,” we often ask people who come to help with Todd’s care. Those who show particularly deep compassion tend to have their own story of suffering, or they’ve loved and cared for someone who suffered. A stage IV cancer survivor. A disabled parent. A sibling who…

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…