Centogene has published data about the use of lyso-Gb1 (glucosylsphingosine) as a sensitive diagnostic and predictive biomarker for Gaucher disease. The study was published in the open-access MDPI journal, Diagnostics, and showed that lyso-Gb1 could be used to predict the clinical course of patients with Gaucher disease. Gaucher disease is a rare inherited metabolic disorder caused by mutations in the beta-glucocerebrosidase (GBA1) gene, responsible for producing a fat-breaking enzyme, glucocerebrosidase. Decreased activity of the enzyme causes fat accumulation in the cells causing symptoms such as liver enlargement, anaemia, bone pain, and spleen enlargement. Multiple biomarkers have been identified for the disease. However, these were present in other diseases and not exclusively in Gaucher disease. Lyso-Gb1 is a more specific biomarker for Gaucher’s, and it can be used to both diagnose the disease and monitor its progression. The confirmation of the biomarker serves as an important step forward in rare disease ...
Sage Therapeutics will reduce its workforce by 40% following the US Food and Drug Administration’s (FDA) rejection of its drug Zurzuvae (zuranolone) in adults with major depressive disorder (MDD). The US-based company will pause some undisclosed early-stage programs and focus instead on the refinement of its compounds SAGE-718 and SAGE-324, per the 31 August announcement. Sage’s leadership will also change with the departure of the current chief scientific officer (CSO) Al Robichaud and the chief development officer (CDO) Jim Doherty. The restructuring comes at a time when the company plans the commercial launch of Zurzuvae for treating postpartum depression which is expected in late 2023. On 4 August, the FDA approved Zurzuvae’s use in women with postpartum depression while simultaneously issuing a complete response letter (CRL) for the treatment’s use in MDD. The CRL stated that the drug’s application did not provide substantial evidence of effectiveness in MDD and that ...
The US Department of Defense (DoD) has awarded PureTech Health $11.4m to help target Fragile X-associated tremor/ataxia syndrome (FXTAS), a late-onset neurological condition that is difficult to diagnose and currently has no effective treatments. Based in Massachusetts, US, Puretech Health will use the funds to advance LYT-300 in a Phase II trial. LYT-300 is an oral prodrug of allopregnanolone, which is known to regulate mood via its positive allosteric modulation of gamma-aminobutyric-acid type A (GABAA) receptors. It is already approved by the US Food and Drug Administration (FDA) as an infusion treatment for postpartum depression. PureTech Health said administration limitations have previously restricted the wider use of the neurosteroid. Developed using the company’s Glyph platform that harnesses natural lipid absorption and transport processes, LYT-300 can be orally administered. FXTAS occurs in premutation carriers of Fragile X Syndrome. Estimates suggest that 30%-40% of male carriers, and 8%-16% of premutation carriers will ...
Pictured: A silhouette of a woman sitting on the floor with her head in her hands/iStock, simpson33 Neumora Therapeutics is making big moves this week. On Tuesday the young biotech announced the initiation of a Phase III program for its potential depression treatment along with a new CEO to lead the way. The nearly two-year-old startup posted statistically significant results for treating moderate-to-severe major depressive disorder (MDD) in its Phase II trial of navacaprant, a kappa opioid receptor (KOR) antagonist. Initiated by BlackThorn Therapeutics prior to its acquisition by Neumora, the trial was amended to include those more serious MDD patients, the population in which it appears to be most effective. In moderate-to-severe patients, navacaprant had statistically significant results in lowering patients’ 17-item Hamilton Rating Scale for Depression score by 3 points at week 4 and 2.8 points by week 8. Across all patients, which included mildly depressed patients, navacaprant did not achieve ...
US-based amyloid diseases treatment solutions developer AltPep has raised $52.9m in its Series B investment round led by Senator Investment Group. Former investors such as Matrix Capital Management Company and Alexandria Venture Investments, who took part in the firm’s Series A round, joined the latest fundraise along with new backers. The new investors in Series B include Eli Lilly and Company, Partners Investment, and Section 32. As part of the deal, Senator Investment Group’s Rohit Vanjani will join the AltPep board. Besides, Lilly’s senior vice president and chief scientific officer for Neurobiologicals Ronald DeMattos will become a member of the Scientific Advisory Board at AltPep. Vanjani noted: “We believe AltPep is in an excellent position to optimise the value of its unique technology. “With scientific application for both therapeutics and diagnostics in a wide range of amyloid diseases, the potential for the company’s pipeline is vast and extremely promising.” The ...
Many neurological conditions that involve involuntary muscle contractions have long been considered as diseases of the brain. However, both the brain and the spinal cord contain many nerve cells associated with movement. New research, published in Science Translational Medicine, used state-of-the-art mouse genetics to distinguish whether the brain or spinal cord was responsible for the disorganization of movement experienced by dystonia patients. Focusing on the most common inherited form of dystonia called DYT1, UCL scientists confined a genetic mutation to the spinal cord of the mice, while sparing nerve cells in the brain. They discovered that the mice consequently developed signs of dystonia that were remarkably similar to those seen in people with the condition. The researchers also observed how specific nerve cells in the spinal cord were affected through the course of the disease. The team hope that their findings will help towards the development of new treatments. Corresponding ...
Magnetic resonance imaging (MRI) is how we visualize soft, watery tissue that is hard to image with X-rays. But while an MRI provides good enough resolution to spot a brain tumor, it needs to be a lot sharper to visualize microscopic details within the brain that reveal its organization. In a decades-long technical tour de force lead by Duke’s Center for In Vivo Microscopy with colleagues at the University of Tennessee Health Science Center, University of Pennsylvania, University of Pittsburgh and Indiana University, researchers took up the gauntlet and improved the resolution of MRI leading to the sharpest images ever captured of a mouse brain. Coinciding with the 50th anniversary of the first MRI, the researchers generated scans of a mouse brain that are dramatically crisper than a typical clinical MRI for humans, the scientific equivalent of going from a pixelated 8-bit graphic to the hyper-realistic detail of a Chuck Close painting. A single ...
Researchers at UC Davis are the first to report how a specific type of brain cells, known as oligodendrocyte-lineage cells, transfer cell material to neurons in the mouse brain. Their work provides evidence of a coordinated nuclear interaction between these cells and neurons. The study was published today in the Journal of Experimental Medicine. “This novel concept of material transfer to neurons opens new possibilities for understanding brain maturation and finding treatments for neurological conditions, such as Alzheimer’s disease, cerebral palsy, Parkinson’s and Huntington’s disease,” said corresponding author Olga Chechneva. Chechneva is an assistant project scientist at UC Davis Department of Biochemistry and Molecular Medicine and independent principal investigator in the Institute for Pediatric Regenerative Medicine at Shriners Children’s Northern California. What are oligodendrocyte-lineage cells? Oligodendrocyte-lineage cells, also called oligodendroglia, are a type of glial cells found in the central nervous system. From birth onward, these glial cells arise to support neural ...
The possible common pathomechanisms linking multiple sclerosis and post-COVID-19 brain involvement (A). Proposal of a new codename regarding post-COVID-19 cognitive sequelae (B). Credit: Journal of Alzheimer’s Disease Reports (2023). DOI: 10.3233/ADR-220090 Infection with SARS-CoV-2 has a significant impact on cognitive function in patients with preexisting dementia, according to new research published in the Journal of Alzheimer’s Disease Reports. Patients with all subtypes of dementia included in the study experienced rapidly progressive dementia following infection with SARS-CoV-2. Since the first wave of COVID-19, neurologists have noticed both acute and long-term neurological syndromes and neuropsychiatric sequelae of this infectious disease. Insights into the impact of COVID-19 on human cognition has so far remained unclear, with neurologists referring to “brain fog.” A group of researchers driven to gain a better understanding of and dissipate this fog investigated the effects of COVID-19 on cognitive impairment in 14 patients with preexisting dementia (four with Alzheimer’s disease [AD], five with vascular dementia, three ...
Bad dreams are common and rarely are a sign of significant health concerns. However, several new studies have found potentially troubling associations between frequent or persistent nightmares and future cognition. These findings, along with recent insights into a potential intervention, have resulted in the top trending clinical topic this week. Previous investigations have found a link between sleep problems in adulthood, including nightmares, and the onset of neurodegenerative diseases. A recent analysis determined a possible association between distressing dreams during childhood and cognitive health concerns (see Infographic). A prospective, longitudinal analysis used data that included all people born in Britain during a single week in 1958. At age 7 years (in 1965) and 11 years (in 1969), mothers were asked to report whether their child experienced “bad dreams or night terrors” in the past 3 months. Among 6991 children (51% girls), 78.2% never had bad dreams, 17.9% had transient bad ...
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