Clinigen’s Aldesleukin Earns FDA’s Orphan Drug Designation as ALS Treatment

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…

Clusterin, a protein that plays a key role in motor neuron health, and ficolin-3, a protein essential for the activation of immune responses, may be promising biomarkers of amyotrophic lateral sclerosis (ALS), according to a recent study. The study, “Levels of clusterin, CD5L, ficolin-3,…

The EveryLife Foundation for Rare Diseases has launched a nationwide National Burden of Rare Disease Survey to measure the full implications, economic and social, of living with rare disease in the United States. People with rare diseases know that the impacts of such conditions extend beyond just medical…

Long delays between symptom onset and diagnosis are common with amyotrophic lateral sclerosis (ALS), and need not be if general practitioners (GPs) were more aware of the disease and its likely “red flags,” a study suggests. Its researchers highlighted specific “flags,” like difficulties with speech or swallowing or muscle…

After our second child was born — back when my husband, Todd, had an undiagnosed weak left arm — we purchased a used minivan. We found the van on Craigslist and drove two hours to purchase it. Six months later, Todd was diagnosed with ALS. We made plans to…

QurAlis Corporation has licensed preclinical small molecule compounds — designed to treat amyotrophic lateral sclerosis (ALS) — from Eli Lilly and Company, furthering QurAlis’s specialization in ALS treatment development. The investigational molecules are aimed at specifically counteracting a pathological symptom of ALS called excitotoxicity, a process…

For Pat Quinn, co-founder of the ALS Ice Bucket Challenge, everyday communication has become more challenging as his amyotrophic lateral sclerosis (ALS) disease has progressed. But eye-tracking technology has helped him, in a sense, retain his “voice.” In fact, Quinn recounts a time when inadvertently letting his…

Barriers need to be overcome in order to facilitate community engagement programs for people with motor neurone disease (MND), a group of diseases that includes amyotrophic lateral sclerosis (ALS), a new study shows. The study, “Supporting access to activities to enhance well‐being and reduce social…

Patient dosing has finished in the pivotal Phase 3 trial assessing the safety and efficacy of repeat administrations of NurOwn, a cell-based therapy for people with amyotrophic lateral sclerosis (ALS), BrainStorm Cell Therapeutics, the therapy’s developer, announced. The Phase 3 trial (NCT03280056), which enrolled…

Nine months after my husband, Todd, was diagnosed with ALS, he entered a social media contest with a $100,000 grand prize to use to fulfill a dream. Contestants with the most votes advanced to the next round. Todd’s dream was to build a handicapped-accessible home near my parents. The home…