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…
Masitinib in combination with Rilutek (riluzole) slows functional decline of patients with amyotrophic lateral sclerosis (ALS) who have a typical disease progression, according to the final report of AB Science‘s Phase 2/3 clinical trial. At 11 months of treatment with…
Posture impairments in amyotrophic lateral sclerosis (ALS) are linked to deficits in trunk control and in the mechanisms that control the body’s response to surface tilts. Moreover, these abnormalities also correlated to disease severity. Those findings are in the study “Abnormal trunk control determines postural abnormalities in Amyotrophic…
Researchers identified small-molecule compounds that help to prevent the build-up of stress-induced clumps of TDP-43 protein, a hallmark of amyotrophic lateral sclerosis (ALS). These findings, although preliminary, may lead to new therapies for ALS. The work “Small-Molecule Modulation of TDP-43 Recruitment to Stress Granules Prevents Persistent TDP-43 Accumulation in…
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing problems as Alport syndrome. Although she had known for years that something was wrong, the news that David would battle a lifelong rare disease devastated…
The klotho protein may have brain-protecting effects in amyotrophic lateral sclerosis (ALS), according to a study in mice that may become a basis for developing new therapies. The study, “Klotho Is Neuroprotective in the Superoxide Dismutase (SOD1G93A) Mouse Model of ALS,” was published in the Journal…
