Utreloxastat (PTC857), an investigational therapy for people with amyotrophic lateral sclerosis (ALS), was safe, well tolerated, and displayed promising pharmacological properties in healthy people, according to a first-in-human Phase 1 study. Based on these findings, the therapy’s developer, PTC Therapeutics, has launched a Phase 2 trial (NCT05349721)…
Phase 1 Safety Study Clears Path to Test Utreloxastat in ALS Patients
I’ve read dozens, if not more, of the kind of column I’m about to write: first-person narratives that offer advice to one’s younger self, full of lessons learned from difficult life experiences. Before my late husband, Jeff, was diagnosed with ALS, I devoured these columns, always looking for some…
Elevated levels of high-density lipoprotein (HDL) — commonly called the “good” cholesterol — are significantly associated with a poorer survival rate among people with amyotrophic lateral sclerosis (ALS), according to a population-based study in the Netherlands. In contrast, levels of total cholesterol and low-density lipoprotein…
Now that my husband, Todd, is paralyzed and uses a noninvasive ventilator, leaving the house is too hard on him. So when we have a date night, we watch a movie at home. Last weekend, we watched “Good Night Oppy,” a documentary about twin rovers that NASA…
The U.S. Food and Drug Administration (FDA) has announced an advisory committee meeting seeking advice on whether to approve Biogen‘s investigational therapy tofersen for the treatment of amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations. The public meeting, to be held online March 22, by…
Neopharm has acquired exclusive rights to commercialize AMX0035 (sodium phenylbutyrate and taurursodiol) in Israel, Gaza, the West Bank, and to the Palestinian Authority as a treatment of amyotrophic lateral sclerosis (ALS). The company’s agreement with Amylyx Pharmaceuticals makes it responsible for the distribution and marketing of AMX0035…
I always love having a good “aha!” moment, especially when it helps improve how I manage my ALS symptoms. Better yet is when I learn that what I thought was a symptom of ALS was actually an impostor, the result of something called “learned nonuse.” Here’s what happened.
An oral suspension form of Radicava (edaravone), an approved treatment of amyotrophic lateral sclerosis (ALS), was favored for patient reimbursement in Canada by a branch of the Canadian Agency for Drugs and Technologies in Health (CADTH). The opinion issued by the CADTH Canadian Drug Expert Committee recommended that Radicava Oral…
The Muscular Dystrophy Association (MDA) has invested $600,000 in PathMaker Neurosystems to help bring a potential noninvasive neuromodulator for amyotrophic lateral sclerosis (ALS) into an initial clinical trial. Funding through the MDA Venture Philanthropy (MVP) program will support an early feasibility study in a small…
What are your plans for this year? What are they for next month? Will you do something you have always wanted to do? Will you do anything different from your usual way of life? After all, it is yet another new year and we want to have a happy…
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