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.,…
Option Care Enterprises will become a national provider of home infusion services for Radicava (edaravone), the first treatment for amyotrophic lateral sclerosis that the United States has approved in more than 20 years. Bannockburn, Illinois-based Option Care will provide the services under an agreement with Mitsubishi Tanabe Pharma America,…
The Boston prep school that ALS patient Pete Frates attended has recognized him for creating the Ice Bucket Challenge that has generated a tidal wave of funding for research on the disease. St. John’s Prep named Frates, who was diagnosed with amyotrophic lateral sclerosis in 2012 at the age of 27,…
The ALS Association has established a disaster relief fund to help patients and their families who were affected by Hurricane Harvey. Harvey brought strong winds and unprecedented rain that caused widespread flooding in parts of Texas and Louisiana. The Houston area recorded 20-plus inches of rain. A lot of people…
Clumps of the protein FUS, which are a pathological characteristic of ALS and frontotemporal dementia, can be prevented through a biological process called phosphorylation, a new study shows. The study, “Phosphorylation of the FUS low‐complexity domain disrupts phase separation, aggregation, and toxicity,” was published in the EMBO Journal. Phosphorylation…
