PharmaFocus Today – Page 392 – media

Discovery suggests a new way to treat spinal muscular atrophy

Journal reference: Kim, J.-K., et al. (2023). A spinal muscular atrophy modifier implicates the SMN protein in SNARE complex assembly at neuromuscular synapses. Neuron. doi.org/10.1016/j.neuron.2023.02.004 Columbia researchers have discovered how a genetic defect leads to spinal muscular atrophy (SMA), a critical piece of information about the disease that neurologists have been seeking for decades. The discovery suggests a new way to treat SMA-;a devastating childhood motor neuron disease that affects 1 in 6,000 children. In the most severe cases, and when left untreated, children born with SMA die within the first two years of life. The researchers also used their finding to develop an experimental therapy that improved survival in mice with severe SMA by 30-fold, one of the greatest increases seen with any treatment in mouse models of SMA. Why the finding matters Almost all cases of SMA are caused by a mutation in a ...
- Source: drugdu
- 147
- April 19, 2023
Study reveals the risk factors associated with developing long Covid

Journal reference: Tsampasian, V., et al. (2023) Risk Factors Associated With Post−COVID-19 Condition. JAMA Internal Medicine. doi.org/10.1001/jamainternmed.2023.0750. Being vaccinated against Covid halves people’s risk of developing long Covid, according to new research from the University of East Anglia. Long Covid still affects some two million people in the UK, and new research published today reveals the risk factors associated with developing the condition. Overweight people, women, smokers and those over the age of 40 are also more likely to suffer from long Covid according to the study – which includes more than 860,000 patients and is thought to be the largest of its kind. The study also finds that co-morbidities such as asthma, COPD, Type 2 Diabetes, coronary heart disease, immunosuppression, anxiety and depression are also associated with increased risk of long Covid. And patients who are hospitalized during their acute Covid infection are also ...
- Source: drugdu
- 139
- April 19, 2023
Abliva’s NV354 receives ODD from US FDA

Sweden-based Abliva has announced that its drug candidate NV354 has been received orphan drug designation (ODD) from the US FDA Office of Orphan Products Development to treat mitochondrial disease. Sweden-based Abliva has announced that its drug candidate NV354 has been received orphan drug designation (ODD) from the US FDA Office of Orphan Products Development to treat mitochondrial disease. Abliva is a clinical-stage company developing medicines to treat rare and severe primary mitochondrial diseases. NV354 is an orally available compound being developed to treat severe primary mitochondrial diseases including Leigh syndrome. Leigh syndrome is a rare disease mainly impacting the central nervous system. Abliva CEO Ellen Donnelly said: “The granting of ODD to NV354 by the U.S. FDA is a validation of the quality of the NV354 pre-clinical program and another important milestone for Abliva. The ODD will be beneficial to us as we work to progress ...
- Source: drugdu
- 124
- April 19, 2023
FDA gives approval to cell therapy Omisirge for blood cancer patients

The US Food and Drug Administration has granted approval to Gamida Cell for Omisirge (omidubicel-onlv), a substantially modified allogeneic (donor) cord blood-based cell therapy. This therapy helps to pace up the recovery of neutrophils (a subset of white blood cells) in the body and lower risk infection. Omisirge is intended for use in adults and pediatric patients aged 12 years and above with blood cancers planned for umbilical cord blood transplantation after a myeloablative conditioning regimen i.e. treatment such as radiation or chemotherapy). US FDA’s Center for Biologics Evaluation and Research director Peter Marks said: “Today’s approval is an important advance in cell therapy treatment in patients with blood cancers. “Hastening the return of the body’s white blood cells can reduce the possibility of serious or overwhelming infection associated with stem cell transplantation. This approval reflects the FDA’s continued commitment to supporting development of innovative ...
- Source: drugdu
- 116
- April 19, 2023
EPA proposes new ethylene oxide rules for medical device sterilization

April 12, 2023 By Jim Hammerand Leave a Comment FacebookTwitterLinkedIn New ethylene oxide rules from the EPA would reduce EtO emissions from commercial sterilizers by 80%, the agency said. The EPA this week proposed new regulations for companies that sterilize medical devices with EtO, following up on last week’s proposal for new EtO rules on chemical plants that make or store the sterilant. EtO is the most common method of medical device sterilization, used for around half of the 40 billion devices sterilized each year in the U.S. For some devices, there’s no alternative to EtO because gamma ray and electron beam sterilization can only be used on certain materials. FAQ: What is electron beam sterilization? But health risks for sterilization plant workers and neighbors have the EPA, FDA, Centers for Disease Control and Prevention (CDC) and Occupational Safety and Health Administration (OSHA) working together to reduce those risks without reducing sterilization capacity. “OSHA’s mission is to ...
- Source: drugdu
- 257
- April 19, 2023
Odds of Heart Attack Six Times Higher With Flu Diagnosis

Marcia Frellick April 17, 2023 People diagnosed with influenza are six times more likely to have a heart attack within the first week that they test positive for the influenza virus than they are in the year before or the year after, a new study indicates. This work, led by Annemarijn de Boer, PhD, with the Julius Center for Life Sciences and Primary Care, UMC Utrecht in Utrecht, the Netherlands, comes 5 years after a 2018 study by Canadian researchers found a similar strong connection between flu and heart attack in people hospitalized for heart attacks. Annemarijn de Boer, PhD The current findings will be presented by de Boer at the at this year’s European Congress of Clinical Microbiology & Infectious Diseases in Copenhagen, Denmark, on April 18. de Boer’s team explains that the connection between influenza and heart attack lies in the influenza virus’s ability to increase the stickiness or clotting of blood. ...
- Source: drugdu
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- April 19, 2023
Higher intake of flavonols linked to lower risk of frailty onset in adults

In a recent study published in The American Journal of Clinical Nutrition, researchers assessed the impact of consuming dietary flavanols on the likelihood of developing frailty. Frailty affects 10% to 15% of older persons and is causally associated with various age-related physiological changes. The need for research in the prevention and treatment of frailty is highlighted by the absence of effective therapies for frailty. Identifying risk factors associated with frailty is crucial for creating interventions that can delay, undo, or prevent its onset. A meta-analysis of several studies found that following a healthy dietary pattern may reduce the risk of frailty onset by 50% to 70%. Flavonoids have the potential to reduce inflammation and frailty development by mitigating the accumulation of oxidative stress and targeting the reduction of age-related senescent cells. About the study In the present study, researchers investigated the relationship between dietary flavonoids, including their subclasses ...
- Source: drugdu
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- April 19, 2023
Binocular visual stimulation followed by sleep helps treat common vision problem in children

Amblyopia, sometimes called lazy eye, is a common vision problem in children and babies, and it’s typically been treated by having the child wear a patch on the stronger eye, with the goal of improving sight for the weaker eye. Recent research has suggested that treatments that require the two eyes to work together might help those with amblyopia recover better. Other research has shown that appropriately timed sleep can help neural networks in the brain repair or restructure themselves, particularly in children, says University of Michigan researcher Sara Aton. Aton’s lab looked at the two research questions in concert, and found that visual stimuli presented to both eyes, rather than the weaker eye alone, helped mice with amblyopia experience a more complete recovery of their visual function. For even better results, the mice had to be allowed to sleep right after their enriched visual experience. The team’s ...
- Source: drugdu
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- April 19, 2023
Brain images just got 64 million times sharper

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 ...
- Source: drugdu
- 136
- April 18, 2023
Researchers discover how some brain cells transfer material to neurons in mice

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 ...
- Source: drugdu
- 155
- April 18, 2023

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