The U.S. Food and Drug Administration (FDA) granted orphan drug designation to AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS), Amylyx Pharmaceuticals, the drug developer, announced in a press release. Orphan drug status aims to encourage therapies for rare and serious diseases through incentives such as seven years of market…
A mechanism that cells normally use to keep them healthy was found to have a dual role in amyotrophic lateral sclerosis (ALS). While the process, called autophagy, delays disease progression in early stages, it contributes to ALS’s deadly spread through the spinal cord in later stages. This study, “…
Neuroscientist and professor Niels Birbaumer, a senior research fellow at Switzerland’s Wyss Center in Geneva, has written a new book investigating the brain’s seemingly limitless capacity to reshape itself and overcome disease. Birbaumer has conducted research with amyotrophic lateral sclerosis (ALS) patients who, because of their progressive motor neuron disease, have…
Severe DNA damage and other disease-associated hallmarks are absent in a mouse model of familial amyothrophic lateral sclerosis (fALS), a new study shows. The study, “DNA strand breaks and TDP-43 mislocation are absent in the murine hSOD1G93A model of amyotrophic lateral sclerosis in vivo and in vitro,” appeared in the journal…
The investigational gene therapy VM202, developed by VM BioPharma and intended for the treatment of amyotrophic lateral sclerosis (ALS), has been shown to be safe and well-tolerated in a small Phase 1/2 clinical trial, according to data published in the journal Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration. While the study,…
The National Institutes of Health has awarded $30 million over two years to 13 researchers who are developing three-dimensional models of amyotrophic lateral sclerosis and other diseases. Researchers say the initiative is aimed at tackling a translational science problem: More than 60 percent of therapies prove ineffective in clinical trials of…
Scientists have discovered how adult skin cells can be converted into motor neurons without transitioning them through a stem cell state. The findings promise to help researchers better understand motor neuron diseases, such as amyotrophic lateral sclerosis (ALS). The study, “MicroRNAs Induce a Permissive Chromatin Environment that…
Project ALS and Amylyx Pharmaceuticals will collaborate on preclinical studies of Amylyx’s lead compound AMX0035 as a potential treatment for patients with amyotrophic lateral sclerosis (ALS). These studies will complement Amylyx’s recently initiated Phase 2 CENTAUR clinical trial (NCT03127514), which will evaluate AMX0035 in 132 patients. Since mitochondrial…
Expression of the calcium channel Cacophony in certain motor neurons restored motor defects in a fruit fly model with TDP-43 loss, a condition found in neurodegenerative diseases like amyotrophic lateral sclerosis (ALS) and frontotemporal dementia, a new study shows. The study, “Restoration of motor defects caused by loss…
The ALS Association of Georgia has received a $10,000 donation from William Mills Agency (WMA), an independent public relations and content marketing firm, to help the non-profit fund research and services to the local amyotrophic lateral sclerosis (ALS) community. William Mills lost its founder, William ‘Bill’ Mills Jr.,…
