uniQure Acquires Rights to Develop APB-102

uniQure has entered an agreement to acquire the full rights to Apic Bio‘s investigational gene therapy APB-102, which is being developed for amyotrophic lateral sclerosis (ALS) associated with SOD1 mutations. uniQure now is planning to start a Phase 1/2 clinical trial of APB-102 later this year. Under the…

Cytokinetics has awarded $20,000 grants to five patient advocacy organizations, including two serving the amyotrophic lateral sclerosis (ALS) community, under its fifth annual Communications Grant Program. Each grant is intended to support the nonprofit’s outreach, engagement, and awareness efforts, allowing the organizations to broaden their work in underserved…

A new pig model of amyotrophic lateral sclerosis (ALS) more accurately reflects the gradual motor neuron loss and disease progression seen in patients, and it may speed the development of ALS treatments, included gene and cell-based therapies, researchers report. The model carries a mutation in the SOD1 gene, an…

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)…

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.