Transthyretin-related cardiac amyloidosis is a progressive disease characterized by the deposition of amyloid protein fibrils in the heart. Amyloid fibril deposition thickens and stiffens the heart walls, and the disease is also known as stiff-heart syndrome. The accumulation of amyloid fibrils causes heart failure, and patients suffer from fluid retention, fatigue, and arrhythmias. The disease can be caused by genetic mutations or related to aging. Prognosis is poor, and untreated patients survive for an average of just 3 years. Now, the results of a study published in the The New England Journal of Medicine (NEJM) promise to radically alter the prospects of patients with this disease. The study was led by Dr. Pablo Garcia-Pavía, who heads the Inherited Cardiac Diseases Section at Hospital Universitario Puerta de Hierro and is a research scientist at the Centro Nacional de Investigaciones Cardiovasculares (CNIC) and within the Spanish cardiovascular research network (CIBERCV). Coinciding with ...
People with strong legs are less likely to develop heart failure after a heart attack, according to research presented today at Heart Failure 2023, a scientific congress of the European Society of Cardiology (ESC). Myocardial infarction is the most common cause of heart failure, with around 6–9% of heart attack patients going on to develop the condition. Previous research has shown that having strong quadriceps is associated with a lower risk of death in patients with coronary artery disease. This study tested the hypothesis that leg strength is associated with a lower risk of developing heart failure after acute myocardial infarction. The study included 932 patients hospitalized in 2007 to 2020 with acute myocardial infarction who did not have heart failure prior to the admission and did not develop heart failure complications during their hospital stay. The median age was 66 years and 753 participants (81%) were men. Maximal quadriceps ...
An international research team led by the German University of Jena has now developed a promising approach to significantly reducing blood clotting on the heart valve material titanium. Around 25,000 artificial heart valves are implanted in Germany per year because the original heart valve is damaged, for example, by an infection. The mechanical heart valves are made of titanium dioxide, among other materials, and last for many years. However, because blood tends to clot on contact with these material surfaces, there is a risk of blood clots forming on the surface of mechanical heart valves. This can become life-threatening if such blood clots break away from the materials. For this reason, most people with mechanical heart valves take medication throughout their lives to reduce blood clotting. An international research team has now developed a promising approach to significantly reducing blood clotting on the heart valve material titanium. The results of ...
The therapy is a brain-penetrant used in various combinations in patients with progressive glioblastoma Debiopharm – a company focusing on future standards of cancer care and infectious diseases – has announced the beginning of its open-label phase 1/2 research of its candidate Debio 0123. The therapy is a brain-penetrant and selective WEE1 inhibitor, used in combination with temozolomide (TMZ) in patients with recurrent or progressive glioblastoma (GBM). It is also being studied in combination with TMZ/RT (SOC) in newly diagnosed patients. The commencement phase of the clinical study aims to establish the recommended phase 2 doses of the treatment. Ultimately, Debiopharm is aiming to meet the pressing requirement for novel treatment interventions to boost quality of life and clinical outcomes for patients living with GBM. Among cancer cells, DNA damage response (DDR) pathways are often upregulated due to genomic instability, increasing the chances of resistance to DNA-damaging therapies. Blocking DNA ...
The US Food and Drug Administration (FDA) has accepted Ardelyx’s resubmitted New Drug Application (NDA) for XPHOZAH (tenapanor) to control serum phosphate in adult patients with chronic kidney disease on dialysis who have had an insufficient response or intolerance to a phosphate binder therapy. The regulatory agency has determined that the NDA is a class 2 review, which leads to a six-month review period from the date of resubmission. It has set a user fee goal date of 17 October this year. The biopharmaceutical company expects XPHOZAH to be commercially available in Q4 2023 after an approval from the regulatory body. Ardelyx president and CEO Mike Raab said: “The acceptance of our NDA is a significant milestone in our journey to bring XPHOZAH to patients. We are excited about the prospect of working collaboratively with the FDA to finalise this review over the next few months. “We are now in ...
As early signs point to a potential resurgence of mpox cases in the U.S. this summer, the Centers for Disease Control and Prevention (CDC) is rolling out real-world data for Bavarian Nordic’s vaccine and urging at-risk people to take two shots.Bavarian Nordic’s Jynneos, given in two doses, is estimated to be 85.9% effective against mpox, a real-world case-match study conducted in 12 U.S. jurisdictions showed. At one dose, the vaccine was found to be 75.2% effective. The CDC released the results in its new Morbidity and Mortality Weekly Report (MMWR). The report comes as a cluster of 21 mpox cases was reported in Chicago, with several diagnoses among those who were vaccinated raising concerns over Jynneos’ efficacy. Although Jynneos’ efficacy may look similar between the partial and full doses in this study of about 900 people, the CDC is still calling for at-risk people to take two doses because the ...
Even after Congress and the Biden administration passed the Inflation Reduction Act (IRA) last summer, some states are looking to further strengthen their drug pricing laws.This week, Minnesota’s legislature endorsed a proposal to create a statewide Prescription Drug Affordability Board. The board would have the power to set upper payment limits on certain high-cost pharmaceuticals, local newspaper Red Lake Nation News reports. The bill also includes provisions to stop price gouging on generics. It’s now on its way to Minnesota Governor Tim Walz for a signature. But that’s where the pharmaceutical industry’s lobby group is hoping to stop the proposal. Wednesday, the Pharmaceutical Research and Manufacturers of America (PhRMA) called on the governor to not sign the bill. The board would “do little to help patients and likely harm future access to medicines,” PhRMA’s senior vice president of state advocacy, Scott LaGanga, said in a statement. “This is a bad ...
In developing immunology treatment Rinvoq, AbbVie is employing the same game plan it used to make predecessor Humira a megablockbuster. And the company seems to be getting better at it.Thursday, AbbVie scored its seventh FDA approval for Rinvoq in a span of less than four years, getting the JAK inhibitor across the finish line as a treatment for Crohn’s disease. A look at Humira’s approval history shows that AbbVie has become more efficient in adding indications to its immunology blockbusters. After Humira was initially blessed in 2002, it took AbbVie 10 years to get the TNF blocker endorsed for its seventh disease type. Coincidentally, Rinvoq’s new FDA approval came hours after the U.K.’s National Institute for Health and Care Excellence signed off on the drug for Crohn’s disease. This is the second gastroenterology indication for Rinvoq after it won approval for ulcerative colitis in March of last year. Rinvoq’s first ...
Adonis — a New York-based startup seeking to create better, more reliable revenue outcomes for providers across the country — has gained capital from some major investors since it launched last year. On Wednesday, the company announced the close of a $17.3 million Series A funding round led by General Catalyst. The round — which brings the startup’s total funding to nearly $23 million — also included investments from Homebrew, Bling Capital and Max Ventures. Adonis, which is a revenue cycle automation platform purpose-built for healthcare, was named after the Greek god known for youthfulness and renewal. “When thinking about the existing state of the revenue cycle, we realized there was dire need for refreshed thinking and a new perspective,” said Adonis CEO Akash Magoon in a recent interview. “Adonis was founded as an organization that would take a refreshed approach to solving some of the biggest administrative burdens through ...
Dermavant Sciences has reported positive results from a phase 3 trial of its plaque psoriasis-approved Vtama (tapinarof) cream in adults and children aged as young as two years with moderate to severe atopic dermatitis. More commonly referred to as eczema, atopic dermatitis is one of the most common inflammatory skin diseases, affecting over 26 million people in the US alone and up to 30% of children worldwide. The disease causes itchy, red, swollen and cracked skin, usually affecting the folds of the arms, back of the knees, hands, face and neck. Dermavant’s ADORING 1 trial met its primary endpoint, with 45.4% of Vtama-treated patients experiencing improvements in an investigator assessment of lesions. This is compared to 13.9% of patients who received vehicle cream. Benefits were also seen across key secondary endpoints, including 55.8% of patients experiencing improvements on the EASI test, which is used to measure the extent and severity ...
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