The US Food and Drug Administration (FDA) has approved Blueprint Medicines’ Ayvakit (avapritinib) as the first and only medicine designed to treat indolent systemic mastocytosis (ISM). Systemic mastocytosis (SM) is a rare haematologic disorder that can lead to a range of debilitating symptoms across multiple organ systems. ISM accounts for the vast majority of SM cases and, despite the availability of multiple supportive care treatments, a number of ISM patients continue to experience a substantial disease burden. Ayvakit, which has been approved in the US to treat advanced SM since June 2021, works by targeting the primary underlying cause of the disease – a mutation known as KIT D816V. Blueprint’s chief medical officer, Becker Hewes, said the approval marked “a shift in the treatment paradigm from supportive care to disease modifying therapy” for ISM patients. “With a broad indication for ISM and a strong label, we are now engaging healthcare ...
Bristol Myers Squibb’s Reblozyl could be an effective initial treatment for anemia patients who are suffering from a group of blood cancers called myelodysplastic syndromes (MDS) and are at a relatively lower risk of progression. That’s the conclusion reached by Olatoyosi Odenike, M.D., from the University of Chicago Medical Center, after she reviewed data from the phase 3 COMMANDS trial that are slated to be presented at the 2023 American Society of Clinical Oncology (ASCO) annual meeting. Odenike is an invited ASCO expert and an opinion leader in the MDS field. The COMMANDS trial is the first study in decades that has shown a benefit for a new agent against erythropoiesis-stimulating agents (ESAs) in low- to intermediate-risk MDS, Noah Berkowitz, M.D., Ph.D., senior vice president of hematology development at BMS, said in an interview with Fierce Pharma. If the FDA eventually agrees with Odenike’s finding, Reblozyl could move from behind ...
A whole host of epilepsy organizations and several members of Congress have come together to call for more governmental help to treat and educate those with epilepsy. The groups, which include the Epilepsy Foundation, the Epilepsies Action Network, CURE Epilepsy, the Rare Epilepsy Network and DEE-P Connections, along with Reps. Greg Murphy, M.D., R-North Carolina, and Jim Costa, D-California, took to Capitol Hill this month to speak about their personal connections to epilepsy. “My son developed epilepsy when he was 14, and it took us months to get the right treatment for him,” said Murphy in a press release. “While he is now 28 and his seizures are controlled, there are still millions of Americans who continue to struggle with seizures. “My sister is one of those people,” he added. “I am committed to elevating the community’s voice in Washington to bring much-needed education and awareness about this very common ...
By James FitzGerald BBC News Elon Musk’s brain-chip firm says it has received approval from the US Food and Drugs Administration (FDA) to conduct its first tests on humans. The billionaire’s Neuralink implant company wants to help restore people’s vision and mobility by connecting brains with computers. It says it does not have immediate plans to start recruiting participants. Mr Musk’s previous ambitions to begin tests came to nothing. The regulator itself is yet to comment. An earlier bid by Neuralink to win FDA approval was rejected on safety grounds, according to a report in March by the Reuters news agency that cited multiple current and former employees. • What is Neuralink? Neuralink hopes to use its microchips to treat conditions such as paralysis and blindness, and to help certain disabled people use computers and mobile technology. The chips – which have been tested in monkeys – are designed to ...
Nicole DeFeudis https://endpts.com/ Editor While net prices for Janssen’s commercial drugs have declined for the sixth consecutive year, out-of-pocket costs have continued to rise for some patients, according to the company’s latest US Pricing Transparency Report. The company provided $39 billion last year in the form of rebates, discounts and fees to commercial health insurers, pharmacy benefit managers and other intermediaries, it said in the report — a more than $5 billion increase compared to 2021. A majority of that, or $11.2 billion, went to commercial payers and PBMs, while 16% (or $6.2 billion) went to the 340B program, 13% ($5.1 billion) went to Medicare and 10% ($3.8 billion) to Medicaid. In 2021, Janssen provided $6.4 billion to the 340B program, $4.6 billion to Medicare and $3.4 billion to Medicaid. “Unfortunately, the reality for millions of patients is growing affordability and health equity gaps caused by underinsurance and inadequate insurance ...
My niece is living in my home while she is attending college. I’ve invited her to eat meals with our family, but she typically declines, desiring to eat alone in her room. I’m concerned because she often seems to eat a lot of food at one time, like two sandwiches with two bags of chips or a quart of ice cream. Could this be a sign of an eating disorder? If so, what can I do to help her? ANSWER: Most people eat too much occasionally. They may fill up an extra plate with seconds at a special event, enjoy another piece of pie at a holiday gathering or eat popcorn until stuffed at the movies. There’s no reason to be concerned if this happens occasionally. But, for some people, overeating can become excessive. It is possible that your niece is suffering from a binge-eating disorder if her excessive eating ...
About 10% of people appear to suffer long COVID after an omicron infection, a lower estimate than earlier in the pandemic, according to a study of nearly 10,000 Americans that aims to help unravel the mysterious condition. Early findings from the National Institutes of Health’s study highlight a dozen symptoms that most distinguish long COVID, the catchall term for the sometimes debilitating health problems that can last for months or years after even a mild case of COVID-19. Millions worldwide have had long COVID, with dozens of widely varying symptoms including fatigue and brain fog. Scientists still don’t know what causes it, why it only strikes some people, how to treat it -– or even how to best diagnose it. Better defining the condition is key for research to get those answers. “Sometimes I hear people say, ‘Oh, everybody’s a little tired,'” said Dr. Leora Horwitz of NYU Langone Health, ...
Dive Brief Boston Scientific has canceled a planned $230 million acquisition of a majority stake in M.I.Tech, a Korean manufacturer of non-vascular stents. The U.S. Federal Trade Commission (FTC) shared news of the cancelation, claiming that Boston Scientific took the action in response to investigations by its staff and overseas enforcement partners. Boston Scientific, which is reportedly considering a $10 billion takeover of cardiovascular device maker ShockWave, agreed to buy a 64% stake in M.I.Tech in June and originally expected to close the deal in the second half of 2022. Dive Insight M.I.Tech is a publicly traded Korean manufacturer of medical devices for endoscopic and urologic procedures, including a line of non-vascular, self-expanding metal stents that Boston Scientific has distributed in Japan since 2015. At the time of the agreement, Boston Scientific framed the products as complementary to its portfolio, and the deal as a way to help M.I.Tech expand ...
1. Use hot water bottles to lower your core temperature Using a hot water bottle might sound like a strange way to combat heat, but James the sleep geek recommends raising your core temperature slightly by placing a luke-warm hot water bottle on your feet thirty minutes before bed; that way, when you go to sleep your core temperature will drop. You can also freeze a hot water bottle and take it to bed with you. 2. Use separate covers from your sleeping partner Use different covers if you are sleeping with a partner; although it might still be tempting for some to snuggle up to your significant other, their body heat will only transfer to you, so use separate covers and if you can and swap out your duvet for a thin cotton sheet. 3. Avoid daytime naps Hot weather can make us sleepy; as tempting as it is ...
Northwestern Medicine scientists have uncovered how peptides produced by bones during inflammation prevent anemia in mice, according to a recent study published in the journal Blood. Inflammation, a feature of many chronic illnesses, is known to lead to the concomitant increased production and cleavage of fibroblast growth factor 23 (FGF23), a hormone previously linked to iron dysregulation. Particularly in patients with chronic kidney disease, the increase of FGF23 is associated with a reduction in iron levels, leading to anemia and increased morbidity, said Valentin David, Ph.D., the Frank Krumlovsky, MD, Professor of Medicine in the Division of Nephrology and Hypertension and senior author of the study. “Intact FGF23 is a phosphaturic hormone mainly secreted by bone cells,” David said. “It was first discovered in 2000, and we have been following it closely since then. We have studied heavily the role of FGF23 in regulating classical mineral metabolism factors, including phosphate and vitamin D. ...
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