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Published on August 16, 2022
Artificial intelligence (AI) has the potential to improve many areas of medicine, with drug development as a key focus. Many companies are now working to apply the power of AI to design better drugs, but six companies are leading the race with candidates already in clinical trials. Over the last…
Published on June 20, 2022
Research led by the University of Exeter and the University of Cambridge in the U.K. adds weight to the theory that motor neurone diseases such as amyotrophic lateral sclerosis (ALS), spinal muscular atrophy, hereditary spastic paraplegias, and others, are caused by abnormal lipid processing. In the current study, published in…
Published on June 15, 2022
This year marks the 10-year anniversary of when CRISPR-Cas9 gene editing was first revealed to the world. Much has happened since then, including wide rollout in many sectors, development of CRISPR-based therapeutics and the discovery that the technology can also be used to develop highly accurate and affordable diagnostics. The…
Published on February 17, 2022
Research carried out by the Genomics England Research Consortium supports the implementation of whole genome sequencing (WGS) for diagnosing neurological repeat expansion disorders. The study, which was published in The Lancet Neurology, showed that WGS was highly accurate at detecting repeat expansions compared with standard tests and even picked up…
Published on January 25, 2022
Researchers were able to suppress a rare aggressive form of amyotrophic lateral sclerosis (ALS) gene using antisense in mice. Their work suggests the therapy could treat cases of the disease caused by mutations in the fused in sarcoma (FUS) gene. The study was recently published in Nature Medicine. “The study is…
Published on January 5, 2022
When his wife retrieved the Christmas lights from their garage in mid December, Foghorn Therapeutics President and CEO Adrian Gottschalk recalled, the decorations she unpacked presented an apt analogy for explaining the science behind his company, a developer of precision therapies. Just as the string of lights was tangled up…
Published on December 7, 2021
A study headed by Cleveland Clinic researchers has identified sildenafil, an FDA-approved therapy for erectile dysfunction (Viagra), and for pulmonary hypertension (Ravatio), as a promising potential drug candidate for helping to prevent and treat Alzheimer’s disease (AD). Research lead Feixiong Cheng, Ph.D., at the Cleveland Clinic Genomic Medicine Institute, and…
Published on December 6, 2021
AviadoBio has come out of stealth mode to announce the raising of an $80M Series A round to develop gene therapies to target neurodegenerative conditions such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The round was co-led by international VC’s New Enterprise Associates and Monograph Capital. Other participants…
Published on November 22, 2021
A drug approved to treat amyotrophic lateral sclerosis (ALS), riluzole, could slow the gradual loss of Purkinje neurons that occurs in Niemann-Pick disease type C1 (NPC1), according to a laboratory study by scientists at the National Institutes of Health (NIH). Mice with NPC1 survived 12% longer when treated with riluzole, compared…
Published on November 15, 2021
Research led by Johns Hopkins University School of Medicine shows high levels of arachidonic acid are present in people and animals with amyotrophic lateral sclerosis (ALS) and that reducing these levels could have therapeutic benefits for people with this devastating condition. The team found that spinal motor neurons in people…
Published on October 19, 2021
In the 1600s, Dutch microscope pioneer Antonie van Leeuwenhoek found various microbes—bacteria, fungi, and other microorganisms—on and in humans. Little did he know that nearly 350 years later, the collection of human microbes would be recognized as a key ingredient in human health. Today, this microbiome plays a role in…
Published on October 14, 2021
Cell therapy biotech Immusoft will partner Takeda to develop new cell therapies to target rare inherited metabolic disorders with central nervous system (CNS) manifestations and complications in a deal that could be worth more than $900M if all targets are met. Immusoft was founded in 2009 and is headquartered in…
Published on July 9, 2021
Researchers based at Washington University School of Medicine have created a genomic atlas of proteins found in the brain of Alzheimer’s disease patients and found new potential drug targets for treating Alzheimer’s, Parkinson’s disease, amyotrophic lateral sclerosis (ALS) and stroke. The team says they have analyzed existing drug databases and…
Published on June 1, 2021
Research reveals a variant in the SPLTC1 gene that causes an early onset form of ALS, as well as a molecular pathway that could explain the neurodegeneration seen in other forms of the disease. Through an investigation of patients with a rare form of amyotrophic lateral sclerosis (ALS), NIH researchers…
Published on May 7, 2021
Research from the University of Florida shows a link between specific bacterial species and physiological changes characteristic of neurodegenerative diseases such as Alzheimer’s disease, Parkinson’s disease, and amyotrophic lateral sclerosis (ALS). People with neurodegenerative disorders exhibit changes in the bacterial composition of their gut microbiome. However, identifying which bacteria may…