Vanda Pinto, PhD,  science writer—

ET-101, Eikonoklastes Therapeutics’ experimental gene therapy for amyotrophic lateral sclerosis (ALS), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). Orphan drug status is given to investigational therapies with the potential to significantly benefit people with life-threatening or chronically debilitating diseases that affect…

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…

An approved medication for heart-related chest pain reduced the frequency and severity of muscle cramps in people with amyotrophic lateral sclerosis (ALS) in a small pilot study. Called ranolazine, the medication was found to be safe and generally well-tolerated — the clinical trial’s key endpoints, or goals. Gastrointestinal…

Although not thought common, laryngospasm — the sudden contraction of the vocal cords that makes speaking and breathing temporarily difficult — does affect some with amyotrophic lateral sclerosis (ALS), a study reported. Excess saliva irritating the vocal cords was the most notable trigger for laryngospasm in their work, the researchers…

AI Therapeutics has launched a Phase 2 clinical trial into AIT-101 (apilimod dimesylate) as a potential oral treatment for amyotrophic lateral sclerosis (ALS) associated with C9orf72 mutations. The trial (NCT05163886) is currently recruiting at the Massachusetts General Hospital and is expected to soon open at sites in…

A Phase 2 study of the investigational antibody AT-1501 in treating amyotrophic lateral sclerosis (ALS) has completed patient enrollment in three of its four dosing groups, and is likely to be fully enrolled by year’s end, its developer, Eledon Pharmaceuticals, announced. A total of 54 ALS patients diagnosed in the…

Early trials in healthy adults supported the safety and tolerability of two potential oral therapies for amyotrophic lateral sclerosis (ALS) — DNL343 to prevent the cellular stress that can promote toxic protein granules, and DNL788 to block a protein linked to brain inflammation — Denali Therapeutics reported. A Phase…

Note: This story was updated Sept. 21, 2021, to note that 18% of patients, not 80%, experienced increases in the MUNIX(4) sum score at some point during the study. The last patient visit has been completed in Clene’s Phase 2 trial investigating the potential of CNM-Au8 — which…

A first patient has been dosed in a Phase 2 clinical trial testing Alector’s investigational antibody AL001 as a treatment for amyotrophic lateral sclerosis (ALS) associated with C9orf72 mutations. The trial is expected to enroll an estimated 45 ALS patients, who will be randomly assigned to AL001 or a placebo,…

Clene, and its wholly owned subsidiary Clene Nanomedicine, have signed two lease agreements that will more than quadruple the manufacturing capacity of CNM-Au8, their investigational therapy for people with amyotrophic lateral sclerosis (ALS). The first lease is for a 10-year agreement for a 74,210-square-foot building in Elkton, Maryland.