News

AC Immune has been awarded more than $500,000 in nonprofit grants by the Michael J. Fox Foundation (MJFF) and the Target ALS Foundation to support its programs to detect abnormal forms of the TDP-43 protein via imaging scans and fluid samples. “It is an honor to have…

A Phase 1/2 clinical trial testing the investigational imaging agent 18F-OP-801 in healthy volunteers and people with amyotrophic lateral sclerosis (ALS) has enrolled its first patient. Ashvattha Therapeutics‘ agent is designed to detect inflammation in imaging exams like positron emission tomography (PET) or MRI scans. This is possible…

Researchers have characterized three molecular subtypes of amyotrophic lateral sclerosis (ALS), each one associated with different clinical outcomes, including age of disease onset, disease duration, and survival. These subtypes are based on unique patterns of gene activity in patient brain tissue and were independent of where the onset of…

A large Phase 3 trial designed to confirm the safety and efficacy of the approved therapy Relyvrio (sodium phenylbutyrate and taurursodiol) for amyotrophic lateral sclerosis (ALS) is now fully enrolled. Amylyx Pharmaceuticals announced the completion of patient recruiting for the trial, called PHOENIX (NCT05021536), which will…

Note: This story were updated Feb. 14, 2023, to correct that ICER analyses found both Radicava ORS and Relyvrio are not cost-effective for the treatment of ALS. The recently approved therapies Relyvrio (sodium phenylbutyrate and taurursodiol) and …

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)…

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…