EU Committee Urges Orphan Drug Status for Clene’s CNM-Au8 for ALS

Orphan drug status has been recommended in the EU for Clene Nanomedicine’s CNM-Au8, an oral therapy candidate for amyotrophic lateral sclerosis (ALS), by a committee of the European Medicines Agency (EMA). CNM-Au8 is emerging as a promising treatment for the progressive neurodegenerative condition, with…

My husband, Todd, has ALS and is paralyzed, so not only do I cook all the meals, but I also feed him. His meals are often rushed or delayed because I’m running our daughter, Sara, to dance practice or wherever else a busy 16-year-old needs to go. There…

A viral protein called HERV-K ENV is often detectable in the fluid around the brains of people with amyotrophic lateral sclerosis (ALS), and is toxic to nerve cells, a new study shows. However, blocking this protein with GeNeuro‘s experimental antibody GNK301 reduced those toxic effects in cell…

The first patient has been dosed in an open-label Phase 2 trial that’s evaluating Seelos Therapeutics’ experimental therapy SLS-005 in people with amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases associated with the accumulation of toxic protein aggregates. Called a basket trial because it tests one therapy in…

Early treatment with Radicava (edaravone) significantly slows disability progression compared with a six-month delay, including in amyotrophic lateral sclerosis (ALS) patients with poorer lung function, according to a new analysis of a Phase 3 clinical trial. Patients switching from a placebo to Radicava after their lung function fell to…

Early use of Clene Nanomedicine’s experimental therapy CNM-Au8 reduces the risk of death by 70% in people with amyotrophic lateral sclerosis (ALS), compared with patients who experienced a nine-month delay in starting treatment. These are the findings of an updated analysis of the RESCUE-ALS Phase 2 trial…

Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world. But a new analysis shows there are as many as 10,867 rare diseases globally. And that…

ABBV-CLS-7262, an experimental compound being developed by Calico Life Sciences and AbbVie for amyotrophic lateral sclerosis (ALS), will likely serve as the sixth therapy in the multi-regimen HEALEY ALS platform trial. HEALEY (NCT04297683) is testing the potential of several ALS treatment candidates simultaneously with a goal of speeding…

A new €2.5 million (about $2.5 million) grant from the European Union (EU) will help advance a prototype vaccine for amyotrophic lateral sclerosis (ALS) associated with mutations in the C9orf72 gene — its most common genetic cause. The European Innovation Council EIC Transition Grant will be used to further develop the vaccine,…

One evening, I took my daily walk in the Nara Nature Park in Houghton, Michigan. As I made my way around a network of wooden boardwalks, through a marsh, and alongside a riverbank and lakeshore, I listened to a symphony of birds. I saw a few couples walking,…