The U.S. Food and Drug Administration (FDA) has approved Mitsubishi Tanabe Pharma America (MTPA)’s oral suspension formulation of edaravone, Radicava ORS, for the treatment of amyotrophic lateral sclerosis (ALS). The oral therapy is designed to offer the same efficacy as MTPA’s Radicava (edaravone) — an FDA-approved, into-the-vein therapy…
In the summer of 2010, my husband, Todd, stopped at the mall after work. It was my birthday, and he hadn’t gotten me anything yet. His mind was still swirling a month after receiving an ALS diagnosis. The shock of it. The grief. He walked through the mall…
A partnership between Rolls Royce and the U.K.-based Motor Neurone Disease (MND) Association is helping people with amyotrophic lateral sclerosis (ALS) preserve the sound of their voices simply by reading a book into a computer. Called voice banking, the process is a way to record a person’s voice…
Silence ALS, a new initiative supported by Target ALS, set as its goals discovering and developing personalized antisense oligonucleotide (ASO) treatments for amyotrophic lateral sclerosis (ALS) patients with rare disease-causing mutations, while advancing understanding of the disease’s overall biology. The initiative, a collaboration between the n-Lorem Foundation, which…
Like many ALS patients who have swallowing issues, a condition known as dysphagia, I follow a regimen of what I can and cannot eat. I’ve accepted this as my new normal for a number of years now, so my emotional reaction to a friend’s recent social media post caught…
A Phase 2 clinical trial has begun dosing patients to evaluate the safety and effectiveness of Denali Therapeutics‘ investigational oral therapy SAR443820 in people with amyotrophic lateral sclerosis (ALS). The study, dubbed HIMALAYA (NCT05237284), is recruiting about 260 adults at sites in the U.S., Belgium, France, Germany,…
ProMIS Neurosciences announced plans to advance a monoclonal antibody against toxic TDP-43 protein clumps as a potential treatment for amyotrophic lateral sclerosis (ALS). The candidate antibody therapy, called PMN267, has shown promise in preclinical experiments conducted at independent institutions, the company also reported in a press release. Nearly all cases…
Treatment with AMX0035 significantly extended the median survival of amyotrophic lateral sclerosis (ALS) patients by more than 10 months compared with a placebo, a new analysis of the Phase 2/3 CENTAUR clinical trial indicates. That survival difference was even greater in patients who continued to receive AMX0035 in the open-label…
A couple weeks ago, one of our toilet tanks developed a crack and started leaking. I turned off the water, and while we waited for a new tank to arrive, we filled a bucket with water from the tub to flush the toilet or used the other bathroom. On Saturday,…
WVE-004, Wave Life Sciences‘ experimental therapy for people with amyotrophic lateral sclerosis (ALS)Â 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…
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