Helixmith has selected Worldwide Clinical Trials to run a planned Phase 2 study of Engensis (VM202), its potential gene therapy for amyotrophic lateral sclerosis (ALS), according to a company press release. Engensis consists of a circular piece of DNA called a plasmid that carries instructions…
PrimeC is safe and well-tolerated for the treatment of amyotrophic lateral sclerosis (ALS) and shows potential to slow disease progression and lung function decline, according to interim results from a Phase 2a clinical trial. Given these results, developer NeuroSense Therapeutics will continue investigating PrimeC with additional analysis…
Newly developed compounds derived from ebselen could be used to treat some types of amyotrophic lateral sclerosis (ALS), a recent study suggests. The study, “Novel Selenium-based compounds with therapeutic potential for SOD1-linked amyotrophic lateral sclerosis,” was published in EBioMedicine. About a fifth of familial…
Patient enrollment is now complete for the Phase 2 clinical trial of Clene Nanomedicine‘s CNM-Au8 as a potentially disease-modifying therapy for amyotrophic lateral sclerosis (ALS). The trial, named RESCUE-ALS (NCT04098406) will test the safety, efficacy, pharmacokinetics, and pharmacodynamics —…
The ALS Association and I AM ALS have opened a petition calling for the earliest possible approval of Amylyx’s experimental oral treatment AMX0035, after a Phase 2/3 trial found the therapy to be safe and to slow functional decline in amyotrophic lateral sclerosis (ALS) patients with rapidly progressing disease. Addressing the…
Younger cycad seeds can contain more toxins than older seeds, and their widespread consumption by people on occupied Guam during World War II could be the environmental origin of the rare sporadic amyotrophic lateral sclerosis (ALS) cluster found there, according to a recent study. Guam residents emerged from their wartime…
A Phase 1 clinical trial of a gene-based therapy for amyotrophic lateral sclerosis (ALS) is now recruiting patients in one of its European centers. The Clinical Research Facility at St James’s Hospital Dublin, in Ireland, has enrolled its first patient and is one of four European centers to participate…
Amylyx’s experimental oral treatment AMX0035 safely and effectively slows functional decline in amyotrophic lateral sclerosis (ALS) patients with rapidly progressing disease, according to full data from the CENTAUR Phase 2/3 trial. Only changes in upper limb strength were significantly different from those seen in placebo-treated patients, the data show. Meanwhile,…
The Phase 3 clinical trial testing an oral suspension formulation of edaravone for amyotrophic lateral sclerosis (ALS) has resumed dosing and patient recruitment, its developer, Mitsubishi Tanabe Pharma America (MTPA), announced. The trial (NCT04165824) was launched in November, but was paused in March due to the COVID-19 pandemic. The study,…
Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…
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