Coya Therapeutics’ ALS001, a potential regulatory T-cell (Treg) therapy designed to halt amyotrophic lateral sclerosis (ALS) progression, has been granted an orphan drug designation by the U.S. Food and Drug Administration (FDA). Furthermore, a Phase 2a trial (NCT04055623) testing ALS001 versus a placebo in 12 ALS patients…
FDA Grants Orphan Drug Status to Coya’s T-cell Therapy ALS001
Using motor neurons derived from people with amyotrophic lateral sclerosis (ALS) is a promising approach to screen for molecules that prevent certain disease mechanisms, and to identify new targets for therapies, according to a recent study. Two classes of therapies were identified that might lessen the excessive firing of motor…
Knowing that so much is riding on me creates a lot of pressure. I don’t get sick days, vacation, or weekends off. Taking time off to attend to my health issues requires finding and paying for caregivers for my husband, Todd, who is paralyzed due to ALS. For example, I…
Strenuous, intense exercise done frequently is an environmental risk factor for amyotrophic lateral sclerosis in people with ALS risk genes, particularly those who carry a faulty C9ORF72 gene, a study concluded. Future research is needed to better understand and identify people at disease risk due to such exercise,…
A July 15 golfing event brings the ALS Therapy Development Institute (ALS TDI) together with he Azzur Group to raise money to help support research into potential new treatments for amyotrophic lateral sclerosis (ALS). The Azzur Fore ALS Golf Tournament is set to take place on Thursday, July…
I’ll admit to hearing voices — the voices in my head, that is. We’re all listening to our mental voices. It’s the constant chatter of inner dialogue or self-talk that leapfrogs through our thoughts, beliefs, questions, and ideas. Mostly the chatter is background noise, accompanying our daily activities and conversations.
Alector and GlaxoSmithKline (GSK) have established a partnership to develop two investigational antibodies — called AL001 and AL101 — to boost the production of the progranulin protein as a potential treatment for neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS). Low levels of progranulin, known as PGRN, which is a…
The first part of Biogen’s early access program for tofersen is now open to patients with the most rapidly progressing cases of familial amyotrophic lateral sclerosis (ALS), the company announced. These patients, with familial ALS caused by mutations in the SOD1 gene, will be the first to…
“I am so sick of being sick,” Todd said. He’s having trouble kicking this bug that he’s been fighting for two weeks. His lungs keep filling with mucus, and I keep clearing them out. Just when we think he’s on the road to recovery because he has a long stretch…
A branch of the European Medicines Agency (EMA) has recommended that Prilenia’s investigational therapy pridopidine be designated an orphan medicine to treat people with amyotrophic lateral sclerosis (ALS). Medicines with the potential to become safe and effective treatments for rare, life-threatening, or chronically debilitating conditions affecting no more than one…
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