WVE-004, Wave Life Sciences‘ experimental therapy for people with amyotrophic lateral sclerosis (ALS) associated with mutations in the C9ORF72 gene, has demonstrated proof-of-concept efficacy in cell and animal models, a new study shows. The nucleic acid-based therapy was able to “potently” reduce the toxic RNA molecules and small proteins…

“Take my hand, precious Lord, Lead me home. … When my life is almost gone, Hear my cry, hear my call, Hold my hand lest I fall.” Although I fall light-years shy of the realm of gospel music aficionado, certain songs of that genre move me like no selection from…

“I been in the right place, but it must have been the wrong time, I’d have said the right thing, but I must have used the wrong line, I’d have took the right road, but I must have took a wrong turn, Would have made the right move, but I…

ALS life sure involves a lot of sitting. Although I keep myself busy with daily projects, most of what I do has me sitting at a computer. Added to that, I sit when eating meals, watching TV, riding in our van, and riding my mobility scooter. That’s a whole lot…

“The secret of life is enjoying the passage of time.”  Time fascinates me, now more than ever. In my own way, in the face of ALS, I’ve learned to enjoy its passing.  I am writing this seconds after hearing…

Occupational exposure to lead — a heavy metal — prior to disease onset is associated with a more severe disease progression and lower survival among people with amyotrophic lateral sclerosis (ALS), according to a recent study. “In survival analysis of an international cohort [group] of ALS cases, our results…

The experimental therapy WVE-004 appears to be engaging its intended target and reducing the amount of toxic proteins in people with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) caused by mutations in the C9orf72 gene, according to early data from the FOCUS-C9 clinical trial. The trial is currently recruiting adults,…

The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…

“‘The problem is all inside your head,’ she said to me, ‘The answer is easy if you take it logically,’ … She said, ‘It grieves me so to see you in such pain, I wish there was something I could do to make you smile again.’ I said, ‘I appreciate…

ALPHA-0602, an investigational gene therapy for amyotrophic lateral sclerosis (ALS), can successfully increase progranulin levels and reduce the toxicity of TDP-43 protein aggregates in cell cultures and mouse models, Alpha Cognition, the therapy’s developer, announced. “These new insights from our preclinical research further support the development of ALPHA-0602…