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Raleigh biotech startup Arrevus gets FDA support for its drug treating cystic fibrosis patients

RALEIGH — Cystic fibrosis (CF) is a heredity disease with no cure. However, Raleigh biotech startup Arrevus believes it owns a drug that could potentially extend the lifespans of those living with the disease — and it’s just got one step closer to making that a reality. Arrevus today announced the U.S. Food and Drug Administration (FDA) has granted Qualified Infectious Disease Product (QIDP) Designation for ARV-1801 (sodium fusidate tablet) for the treatment of pulmonary exacerbations in CF patients. These episodes of “acute worsening of pulmonary status” occur frequently in patients with cystic fibrosis, and result in a sequential and permanent decline in lung function. “We know that ARV-1

Organ Care System Used at Florida Hospital Expected to Make More Lungs Suitable for Transplant

Tampa General Hospital (TGH) announced that is the first in Florida to use an organ care system that allows donor lungs, hearts, and livers to stay viable and working for hours during transport, and is likely to expand the number of life-saving transplants, including lung transplants for people with cystic fibrosis (CF), done at its center. The first lung transplant in Florida using the machine — the Organ Care System by TransMedics — was performed on Oct. 22 at TGH. “We can maintain the organs for longer periods, which means we can retrieve them from a wider geographic area,” John Dunning, MD, surgical director for Heart and Lung Transplantation at TGH, said in a press release. “And, the co

Creating Intestinal Organoids In Vitro

Scientists in Boston say they have developed a new method for generating groups of intestinal cells that can be used, among others, to make disease models in the lab to test treatments for diseases affecting the gastrointestinal system. Using human induced pluripotent stem cells, their approach combined a variety of techniques that enabled the development of three-dimensional groups of intestinal cells (organoids) in vitro, which can expand disease treatment testing in the lab using human cells. The study (“Generation of mesenchyme free intestinal organoids from human induced pluripotent stem cells”), published online in Nature Communications, provides a novel platform to improve drug screen

CF Carriers at Increased Risk for Disease-related Conditions, Study Reports

Cystic fibrosis carriers — individuals who have certain genetic mutations in one of the copies of the CFTR gene — have a significantly increased risk compared with healthy people for several CF-related conditions, a new study shows. The study, “Cystic fibrosis carriers are at increased risk for a wide range of cystic fibrosis-related conditions,” was published in the journal Proceedings of the National Academy of Sciences. Because CF is a recessive genetic disorder, both copies of a person’s CFTR gene — one inherited from the mother and the other from the father — must be mutated to develop the full-blown disease. People with CF develop recurrent pulmonary infections and also hormonal, gastr

SIMPLE TREATMENT OF TYPE 2 DIABETES WITH A LOW-CALORIE DIET IS SO EFFECTIVE THAT IT REVERSES THE DIS

A “revolutionary” treatment plan for type 2 diabetes has made the British National Health Service (NHS) very active as they position themselves to adopt a new standard of treatment. The course of treatment consists of a liquid diet of 800 calories to be taken in the form of a soup or a shake daily for a period determined according to the time elapsed since the patient developed type 2 diabetes. Groundbreaking research was born out of Newcastle University which seems to have stuck a pin in many of our hypotheses about type 2 diabetes, as well as proven almost beyond a shadow of a doubt that type 2 diabetes is actually reversible, especially in new patients. Treatment is essentially a prescrip

5 FDA approval decisions to watch in the 1st quarter

Credit: Yujin Kim / MedTech Dive, original photo courtesy of U.S. Food and Drug Administration The Food and Drug Administration cleared for market 48 new drugs through its main review office last year. Though that's lower than the 59 approvals seen in 2018, the agency's decisions still provided more treatment options for patients living with cystic fibrosis, sickle cell disease and rare muscular disorders. Notably, the agency ended 2019 with a flurry of earlier-than-expected decisions, bolstering the 2018 count with several drugs it was scheduled to finish review on this year. It's unclear how or if the approval stream will change in the new year and decade. The first quarter of 2020, though

Medical Mystery: It took more than 50 years to find out why his health was so poor

For as long as he can remember, Steven Knapp never felt well. “That was pretty much true from early childhood,” said Knapp, a self-described “Army brat” who was born in Germany and grew up in Turkey and a half-dozen states, including Georgia, where he has lived since high school. Now 61, the technology writer said that childhood trips to the emergency room were not uncommon. A small and skinny child, he seemed accident-prone. Once he hit his 30s, Knapp recalled, his health seemed to nose-dive and he suffered unexplained bouts of exhaustion. But invariably when doctors tested him, he said, “my bloodwork was surprisingly good.” One of his most perplexing and bothersome symptoms was a chronic c

New agents to fight multidrug-resistant germs

Resistance to antibiotics is on the rise worldwide. Fraunhofer scientists have joined forces with partners in the Phage4Cure project to explore alternatives to antibiotics. One objective is to vanquish multidrug-resistant pathogens with viruses called bacteriophages. Another is to see these phages approved to treat the dreaded hospital germ Pseudomonas aeruginosa, the most frequent bacterial cause of pneumonia. According to the World Health Organization (WHO), antibiotic resistance has reached alarming levels worldwide. WHO Secretary General Tedros Adhanom Ghebreyesus says it is jeopardizing a hundred years of medical progress. Finding a solution to this problem is one of the most pressing c

CF Newborn Screening Improves Weight and Lung Outcomes, but Has No Impact on Social Inequalities, St

Babies diagnosed with cystic fibrosis (CF) through newborn bloodspot screening show better lung function, can better stave off infection with Pseudomonas aeruginosa (the most common bacteria found in CF), and have better weight outcomes than those diagnosed clinically later in life, a large CF registry-based study from the United Kingdom shows. However, newborn screening had no clear impact on children from disadvantaged backgrounds. The study, “Impact of newborn screening on outcomes and social inequalities in cystic fibrosis: a UK CF registry-based study,” was published in the journal Thorax. Estimates state that one in every 2,500 newborns in the U.K. has CF. Most children are diagnosed s

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