A single dose of an artificial microRNA — a tiny RNA molecule that is able to control the activity of certain genes — can lower the activity of SOD1, a gene commonly mutated in patients with familial amyotrophic lateral sclerosis (ALS), a proof-of-concept study shows. According to researchers,…
Gene Silencing Method Shows Promise to Treat Familial ALS Associated With SOD1 Mutations
Searching for a New Map
“I keep pulling it out — the old map of my inner path,” Joyce Rupp writes in her poem “Old Maps No Longer Work.” I was introduced to Rupp’s poem on a spiritual retreat five years ago. Her words resonated with me then, five years past my husband…
Changes in commonly tested blood biomarkers in people with amyotrophic lateral sclerosis (ALS) may be associated with faster disease progression and an increased risk of mortality, according to a recent study. Given the easy access and low cost of these standard…
“Oh, I used to be disgusted and now I try to be amused.” Those lyrics from the Elvis Costello song “(The Angels Wanna Wear My) Red Shoes” are an apt description of the journey that my perspective…
Tofersen, Biogen’s investigational therapy for people with familial amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations, was safe and generally well-tolerated over three months, and appeared to lower SOD1 protein levels in the central nervous system, a Phase 1/2 clinical trial has found. There…
Let’s Go to the Movies
During these past few months, like most folks I know, I’ve been watching movies — lots of movies. And by now, my wish list of titles is pretty well picked over. So, whenever a new release comes out, I jump on it, thinking “OK, Netflix, surprise me!” Well, I got…
Bereaved caregivers of people with motor neurone disease (MND) are at increased risk of prolonged grief disorder compared to the general bereaved population, a new study suggests. This indicates a need for greater support for bereaved caregivers of people with MND. The study, “Grief, depression, and anxiety…
A comparison of amyotrophic lateral sclerosis (ALS) among U.S. military veterans found those who lived the longest with this disease — an average of 16.3 years in more than 40% of those studied — had a younger age at onset but slower initial progression. Findings…
Low doses of Clinigen’s aldesleukin, an immunotherapy used in certain types of cancer, safely boosted the number and function of regulatory T-cells (Tregs), a type of immune cell that keeps others in check, in people with amyotrophic lateral sclerosis (ALS), according to data from a pivotal Phase 2…
Expanding World, Shrinking World
When I was a child, my elderly great-aunt Martha joined my family every Christmas Eve to participate in gift opening. I felt bad that she only got one present while my brothers and I received so many. But she seemed happy with the pair of slippers or homemade ornaments my…
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