The hexanucleotide repeat expansion (HRE) GGGGCC in the noncoding region of the chromosome 9 open reading frame 72 (C9ORF72) gene is the most common cause of hereditary (40%) and apparently sporadic (5%-6%) amyotrophic lateral sclerosis (ALS).
Quralis Corporation’s clinical trial application (CTA) for QRL-201, a first-in-class Stathmin-2 (STMN2) precision medicine for amyotrophic lateral sclerosis (ALS), has been authorized by Health Canada.
Eikonoklastes Therapeutics Inc. and Forge Biologics Inc. have established a manufacturing partnership to advance Eikonoklastes' adeno-associated viral (AAV)-based gene therapy, ET-101, into clinical trials for the treatment of patients with amyotrophic lateral sclerosis (ALS).
Stimulating the brain via implanted electrodes is used to treat both movement disorders such as Parkinson’s disease, and some psychiatric conditions such as obsessive compulsive disorder. But researchers are also working on ways to make such implanted electrodes listen instead of talk – and translate neuronal signals for people that have lost the ability speak, or the ability to move.
Fundamental Pharma GmbH has raised €10 million (US$10.3 million) in a seed round to develop a new class of glutamate inhibitors, after uncovering a route to maintaining the protective effects of the neurotransmitter in the synapses while preventing neurotoxicity when it is released elsewhere.
NRG Therapeutics Ltd. has closed a £16 million (US$18.3 million) series A round to take forward programs in Parkinson’s disease and amyotrophic lateral sclerosis, after pinning down the target of its brain penetrant small molecules.
Stimulating the brain via implanted electrodes is used to treat both movement disorders such as Parkinson’s disease, and some psychiatric conditions such as obsessive compulsive disorder. But researchers are also working on ways to make such implanted electrodes listen instead of talk – and translate neuronal signals for people that have lost the ability speak, or the ability to move. At the Neurophysiology: Decoding and Neural Processing II session of the 2022 Annual Meeting of the Society for Neuroscience in San Diego, researchers from the Wyss Center for Bio and Neuroengineering (Switzerland) presented a device implanted in the brain that allowed restoration of movement and speech.
The FDA has awarded orphan drug designation to Eikonoklastes Therapeutics Inc.'s ET-101 program for the treatment of amyotrophic lateral sclerosis (ALS).
NRG Therapeutics Ltd. has closed a £16 million (US$18.3 million) series A round to take forward programs in Parkinson’s disease and amyotrophic lateral sclerosis, after pinning down the target of its brain penetrant small molecules.
The wholesale acquisition cost for Amylyx Pharmaceuticals Inc.'s new U.S. FDA-approved amyotrophic lateral sclerosis (ALS) treatment, Relyvrio, has been set at about $158,000 for the first year’s treatment. It jumps to about $163,000 in the second year, the company said, a change that would move it closer to the cost of competitor Mitsubishi Tanabe Pharma Corp.'s Radicava (edaravone), which costs about $165,000 annually. A 28-day prescription will cost $12,504.