The long noncoding RNA MINCR, implicated in ALS and Alzheimer’s disease as well as several types of cancer, appears to function differently when present at high versus low levels.
After six months, patients with fast-progressing amyotrophic lateral sclerosis who had received the experimental treatment had less loss of function than those who received a placebo.
Researchers slowed disease progression in the mice by injecting two different viral vectors, each containing one part of the DNA encoding the Cas9 protein, to edit the causative gene.
Flexible proteins appear to protect molecules from becoming denatured in extreme conditions such as heat and from clumping up, as happens in some neurodegenerative diseases.
Boosting the levels of Akkermansia muciniphila in mouse guts slowed the progression of an ALS-like disease, while two other microbiome members were associated with more severe symptoms.
He developed an MRI-based map of the human cortex, discovered genetic risk factors for neurodegenerative diseases, and wrote about his struggles with ALS.
Amyotrophic lateral sclerosis patients in the U.S. now have an option besides riluzole, which was approved by the FDA more than two decades ago and only extends life by two or three months.
At first blush, do-it-yourself clinical trials seem pointless and reckless. But a deeper truth pervades the research and the patients who drive it forward.
In the midst of a debate about an experimental drug’s early approval, the US Food and Drug Administration requests that full trial results be released.
