Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…
News
Using a recumbent stepper is a safe and well-tolerated way for patients with amyotrophic lateral sclerosis (ALS) to do physical exercise, a pilot study suggests. The study, “Recumbent stepping aerobic exercise in amyotrophic lateral sclerosis: a pilot study,” was published…
GPM Investments raised $244,263 for patients with amyotrophic lateral sclerosis (ALS) and other neuromuscular disorders in its eighth holiday pinup fundraiser in 2018 in partnership the Muscular Dystrophy Association. Approximately 1,400 GPM convenience stores throughout the East Coast and Midwest regions of the U.S. participated in the campaign, including…
A potential therapy for amyotrophic lateral sclerosis (ALS) consisting of an antibody that reduces the abnormal accumulation of the TDP-43 protein improved cognitive and motor performance in mice, a study shows. The study, “Viral-mediated delivery of antibody targeting TAR DNA–binding protein 43 mitigates associated neuropathology,” was published in The…
Warning the reporter accompanying him not to take any pictures, veteran horticulturalist Michael Castleman punches an electronic code and unlocks the door to Room 209, nicknamed the “Mother Room.” Photography is indeed forbidden inside this living vault, which contains 20 phenotypes of cannabis plants thriving under the glare of 25…
A new diagnostic score accounting for age at disease onset and motor neuron dysfunction in the brain is able to distinguish amyotrophic lateral sclerosis (ALS) from other neuromuscular disorders early in disease process, a study reports. The study, “Amyotrophic lateral sclerosis diagnostic index: Toward a personalized diagnosis of ALS,”…
A gene therapy designed to block the activity of SARM1 protein prevented the loss of axons — long projections that connect nerve cells and transport information — making the therapy a potential strategy to reduce the loss of peripheral nerves in several neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS), according to a mouse…
Amyotrophic lateral sclerosis (ALS) patients who complete the ongoing Phase 3 REFALS clinical trial evaluating levosimendan (also known as ODM-109) will soon have the opportunity to enroll in an open-label extension study. Levosimendan is an oral treatment developed by Orion that is being investigated for its ability to relieve breathing…
A small molecule known as mir-494-3p might play a protective role in the survival of motor nerve cells, a discovery that could lead to the development of new therapies for amyotrophic lateral sclerosis (ALS), a study suggests. The study, “Micro-RNAs secreted through astrocyte-derived extracellular vesicles cause neuronal network…
G71.01 is, quite literally, code for Duchenne muscular dystrophy. Likewise, Q93.51 stands for Angelman syndrome, and G40.419 covers generalized and treatment-resistant epilepsies, which groups like Orphanet and the American Epilepsy Society define as including Dravet syndrome. All three designations are among some 70,000 others listed in the…
Recent Posts
- AB Science adds new biomarker to Phase 3 masitinib ALS clinical trial
- ‘Early birds’ have significantly lower ALS risk than ‘night owls’: Study
- As my late husband’s ALS progressed, we kept our bucket list simple
- Tossing and turning over fears this ALS journey has come to its end
- Experimental ALS therapy QRL-201 shows potential to slow disease decline