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,”…
“I get by with a little help from my friends.” Or, in my case, A LOT. Two weeks ago, I discussed the ALS village. Last week, I wrote that I’ve survived 12 years since ALS darkened my doorstep. The latter is not possible without the former.
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…
The loss of a key enzyme, called adenosine deaminase, in astrocytes — the energy-supporting cells of neurons — leads to a toxic accumulation of molecules that contributes to the death of motor neurons seen in amyotrophic lateral sclerosis (ALS), a study shows. The study, “Astrocyte adenosine deaminase loss increases…
The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— how disorders such as spinal muscle atrophy(SMA) run their course if untreated — the choice of “efficacy endpoints” in clinical trials, and how…
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