Patricia Inácio, PhD,  science writer—

Researchers report having created a new human-on-a-chip model, one able to more accurately simulate the clinical features of amyotrophic lateral sclerosis (ALS) in people. The chip uses motor neurons — the nerve cells responsible for controlling voluntary muscles — derived from stem cells isolated from ALS patients. This approach…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Clinigen’s aldesleukin for the treatment of amyotrophic lateral sclerosis (ALS). Aldesleukin is a lab-made version of the interleukin 2 (IL-2), an immune signalling molecule known to play a key role in the maintenance of a…

The levels of certain indicators of iron status, namely ferritin and transferrin, are altered in patients with amyotrophic lateral sclerosis (ALS) relative to healthy controls, suggesting a possible link between iron metabolism and ALS, a study has found. The results, “Abnormal Serum Iron-Status Indicator Changes in…

The pivotal Phase 3 clinical trial testing BrainStorm Cell Therapeutic’s cell-based therapy NurOwn in amyotrophic lateral sclerosis (ALS) patients continues with only occasional scheduling changes to treatment due to the COVID-19 pandemic. Patient dosing is expected to be complete by July, the company announced. “BrainStorm made…

Tikomed has secured $5.5 million in financing from investors to support the ongoing clinical development of ILB, its lead therapy candidate for degenerative neurological diseases, including amyotrophic lateral sclerosis (ALS). The funding also will help develop Ibsolvmir, a therapy to increase the effectiveness of transplanting cells to treat patients…

The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still lacking treatment. And federal incentives to boost treatment development for these rare diseases have primarily focused not on creating new…

The European Medicines Agency (EMA) granted orphan drug status to Cytokinetics‘ reldesemtiv for the treatment of amyotrophic lateral sclerosis (ALS), the company announced. The move follows fast track designation granted in December by the U.S. Food and Drug Administration for the same indication, and provides the…

In the coming months, Alexion Pharmaceuticals will launch a pivotal Phase 3 clinical trial to test its complement system inhibitor Ultomiris (ravulizumab) in people with amyotrophic lateral sclerosis (ALS) whose motor symptoms began in the prior three years. The CHAMPION-ALS trial — which follows an investigational new…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Cytokinetics and Astellas Pharma‘s reldesemtiv for the treatment of amyotrophic lateral sclerosis (ALS), the companies announced. Reldesemtiv, a fast skeletal muscle troponin activator designed to improve muscle function with minimal nerve…

The U.S. Food and Drug Administration has agreed to expand Tiglutik, a liquid oral form of riluzole, to include giving it via a feeding tube to treat amyotrophic lateral sclerosis (ALS), ITF Pharma, the company marketing Tiglutik, announced. The approval of administering the therapy via percutaneous endoscopic…