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,…
WVE-004 Reduces Toxic Proteins, Early FOCUS-C9 Data Suggests
Blocking channels containing the connexin 43 (Cx43) protein on astrocytes — a type of nerve support cell — slowed disease progression in a mouse model of amyotrophic lateral sclerosis (ALS), a study found. The protein was found to be increased in ALS patient tissues and spinal fluid, and its…
My husband, Todd, gets twice-weekly physical therapy, which significantly improves his quality of life with ALS. Six years ago, Todd was having pain in his shoulders, and his elbows would not fully straighten, making it difficult for him to use his wheelchair. His doctor ordered physical and occupational therapy evaluations…
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…
A first healthy volunteer has been enrolled in a Phase 1 trial and given a first dose of PrimeC, an investigational combination therapy for people with amyotrophic lateral sclerosis (ALS), the treatment’s developer, NeuroSense Therapeutics, announced. The open-label Phase 1 trial (NCT05232461) is a pharmacokinetic study,…
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…
Based on data from a Phase 1 clinical trial, Biogen and Ionis Pharmaceuticals are discontinuing development of BIIB078, their experimental treatment candidate for amyotrophic lateral sclerosis (ALS) caused by mutations in the C9orf72 gene. While the therapy was generally safe and well-tolerated in people with C9orf72-associated ALS, it…
Team Drea Foundation is launching a customizable wellness challenge that seeks to raise $50,000 in 50 days for amyotrophic lateral sclerosis (ALS) research. The 50for50 challenge invites people to set a personal goal related to the number 50, such as 50 pushups a day or 50 acts…
I got away for a couple of nights during spring break with my 12-year-old son, Isaac, and 16-year-old daughter, Sara. It was good to have a mini-vacation, although I was sad my husband, Todd, couldn’t join us because his ALS progression makes travel too difficult. Three years ago, we all…
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