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, “…
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…
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…
“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…
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…
Would it surprise you to learn that I check my cellphone zero times a day? All around me, people are texting, emailing, chatting, and checking social media. Me? Nada. My eyes are forward, watching the world. I’m embracing “the joy of missing out” — a state of mind caused by…
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