News

Patient enrollment is now complete, ahead of schedule, for the Phase 3 ORARIALS-01 clinical trial assessing the effectiveness of Orphazyme’s investigational therapy arimoclomol in the treatment of amyotrophic lateral sclerosis (ALS). “The completion of enrollment for Orphazyme’s pivotal ALS trial is another great milestone for our clinical development program. We are confident that…

Mallinckrodt Pharmaceuticals has shut down its Phase 2b PENNANT clinical trial assessing the effectiveness and safety of H.P. Acthar Gel, an experimental injectable therapy for amyotrophic lateral sclerosis (ALS). The reason for this decision was an increased incidence of pneumonia among ALS patients receiving H.P. Acthar Gel compared to…

A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of treatments for the 95% of such illnesses that currently don’t have one. The European Joint Programme on Rare Diseases (EJP…

Specific populations of neurons and associated cells in the spinal cord of people with sporadic amyotrophic lateral sclerosis (ALS) may serve as early biomarkers for the disease, a study has found. The newly discovered populations offer scientists a chance to learn more about the first steps underlying ALS and…

Scientists who dissected the structure, and mechanism of action, of the protein Cdc48 — dubbed the “Swiss Army knife of cells” — say it can be explored as a therapeutic target for several disorders, including amyotrophic lateral sclerosis (ALS). The findings of the study, “Structure of the…

The Phase 3 REFALS trial exploring the impact of oral levosimendan (ODM-109) in patients with amyotrophic lateral sclerosis (ALS) has completed its target enrollment, the therapy’s manufacturer, Orion, announced. A total of 496 participants were recruited across 104 sites in the United States, Canada, Europe, and Australia. They…

A study in identical twins or triplets found that changes in DNA marks, collectively known as DNA methylation and typically associated with aging, can like genetics play an important role to determining the development of amyotrophic lateral sclerosis (ALS). The four sets of ALS-affected and unaffected siblings carried age-related differences…

Researchers have created the first entirely human small chip that is able to replicate some of the cell-cell interactions occurring in the peripheral human nervous system, according to a recent study. The Nerve-on-a-Chip platform, developed by AxoSim using organ-on-chip technology, promises to help researchers understand, faster and at…

Men with benign prostatic hyperplasia (BPH) — prostate gland enlargement — are not at greater risk of amyotrophic lateral sclerosis (ALS) and similar disorders, according to a nationwide study in Denmark. “We found no evidence of an increased risk of ALS and other MND [motor neuron diseases] among…

The challenges Vesna Aleksovska faced when she decided a decade ago to help fellow Macedonians with rare diseases were so daunting, they would have scared off all but the most determined. At that time, few doctors in the developing country of 2 million — now called North Macedonia — had…