Search Results for “EC” – Page 422 – media

Krystal Gets First FDA Approval for Redosable Gene Therapy, Rare Skin Disease

By Tristan Manalac Pictured: Blue sign outside FDA building/JHVEPhoto/Adobe Stock The FDA has approved Krystal Biotech’s topical gene therapy beremagene geperpavec-svdt, now to be marketed as Vyjuvek, for the treatment of the rare skin disease dystrophic epidermolysis bullosa, the company announced Friday. Vyjuvek’s label covers its use in dystrophic epidermolysis bullosa (DEB) patients aged six months and above bearing mutations in the COL7A1 gene. Friday’s approval makes Vyjuvek therapy the first-ever redosable gene therapy and the first DEB therapeutic to hit the market. “Until now, there have been no approved treatment for DEB in the United States, and patients, families and physicians were limited to palliative care,” Andy Orth, chief commercial officer at Krystal Biotech, said in an investor call Friday evening, adding that there are likely around 3,000 DEB patients in the U.S., of whom some 1,100 have confirmed diagnoses, according to claims analytics. The company expects to launch ...
- Source: drugdu
- 111
- May 24, 2023
New clues to why some people suppress HIV without drugs

Some HIV patients are naturally able to keep the virus fully in check without any medicinal help, a phenomenon that has intrigued scientists for decades. New research appears to identify at least one reason why: an abnormally powerful version of an infection-fighting white blood cell called CD8+ T cell. CD8+ T cell’s are a type of T cell, a normal feature in everyone’s immune system. T cells typically amass in the lymph nodes of an HIV-infected patient. But among “spontaneous controllers”—those with the rare ability to automatically corral HIV and prevent it from triggering illness—investigators found that CD8+ T cells appear significantly more adept at identifying and stopping HIV. “About one in 300 people are able to control HIV without the need for medications,” noted study author Dr. Bruce Walker, director of the Ragon Institute of MGH, MIT and Harvard, an immune system research center in Cambridge, Mass. It appears ...
- Source: drugdu
- 210
- May 24, 2023
Blueprint’s Ayvakit picks up long-awaited FDA approval in indolent systemic mastocytosis

In a development that’s set to change the lives of many patients, Blueprint Medicines’ Ayvakit has finally won its long-awaited FDA approval to treat indolent systemic mastocytosis (SM). The drug won its original approval in 2021 to treat the advanced form of the disease, which affects only 5% to 10% of those with SM. Now, Ayvakit can reach the majority of SM patients, who suffer from the disease’s indolent form. The larger patient pool comprises approximately 30,000 U.S. patients, but so far only about half are diagnosed, Blueprint’s chief operating officer Christy Rossi told Fierce Pharma in an interview. Indolent SM patients often end up “living their lives in a bubble,” Rossi said. Physicians can treat symptoms, which range across organ systems from skin lesions to brain fog, but until now there was no option to treat the underlying disease. Enter Ayvakit. The drug targets a mutation called KIT D816V, ...
- Source: drugdu
- 150
- May 24, 2023
Study helps clarify the underlying cause of chest symptoms in angina

An intensive study into the treatment of angina has found that the use of tests for small blood vessel function in the heart clarify the underlying cause of the condition. Patients referred to hospital by their General Practitioner (GP) for assessment of chest pain were invited into the study if a heart scan had ruled out blocked heart arteries. The trial showed that the common underlying cause of chest symptoms in angina sufferers was myocardial ischaemia with no obstructive arteries (INOCA), which wasn’t diagnosed by a standard CT heart scan. The diagnosis was clarified using the tests where INOCA was 4 times more likely, while ‘normal’ results were half as less likely to detect small vessel disease. The study, funded by the British Heart Foundation and the Chief Scientist Office of the Scottish Government and sponsored by the NHS Golden Jubilee, was carried out by heart specialists from the University ...
- Source: drugdu
- 119
- May 23, 2023
Intercept’s NASH dreams may be dashed after FDA panel votes against Ocaliva’s approval bid

It looks like Intercept Pharmaceuticals’ Ocaliva still has a tough road ahead in nonalcoholic steatohepatitis (NASH) after a panel of FDA advisers voiced concerns with the company’s application package. During a one-day meeting of the FDA’s Gastrointestinal Drugs Advisory Committee, panelists voted 12 to 2—with 2 abstentions—that the benefits of Ocaliva 25 mg, also known as Obeticholic acid, or OCA, don’t outweigh the risks in NASH patients with stage 2 or 3 fibrosis. Further, in response to the question about a potential accelerated approval, panelists voted 15 to 1 against that regulatory pathway for the drug. Many of the nay-sayer’s concerns came down to Intercept’s use of a surrogate endpoint in its clinical trial. Panelists’ concerns also centered on the potential for OCA to cause drug-induced liver injury. While the FDA isn’t beholden to the advice of its advisory committees, the regulator often follows its experts’ lead. Intercept’s NASH candidate ...
- Source: drugdu
- 111
- May 23, 2023
Lung association calls for more Black Americans to join clinical trials in pharma-backed effort

Beth Snyder Bulik Senior Editor The American Lung Association is doubling down on efforts to encourage more Black Americans to join lung cancer clinical trials. The new pharma-sponsored “Awareness, Trust and Action” initiative aims to put those three words into action with a national campaign that includes digital posts and social media. The first posts are slated to go up on Saturday, which is Clinical Trials Awareness Day. Daiichi Sankyo, Genentech, Merck, Novartis and Novocure provided financial support for the project. Cedric “Jamie” Rutland “We have to do a better job of recruiting Black Americans into clinical trials and letting them know why,” said pulmonologist Cedric “Jamie” Rutland, a national volunteer medical spokesperson for the American Lung Association. “And not only that, but teaching Black Americans what lung cancer is, what the risk factors are, why they have risk factors living in heavily polluted areas … We have to ...
- Source: drugdu
- 112
- May 23, 2023
Cholera outbreak kills 10 in S.Africa

An outbreak of cholera has killed at least 10 people near South Africa’s capital of Pretoria, health authorities said Sunday, urging the public to be “extra vigilant”. The Department of Health in Pretoria’s Gauteng province said 95 people visited a local hospital since Monday showing cholera symptoms, including diarrhea, stomach cramps and nausea. Lab tests on Sunday confirmed at least 19 were cases of cholera, the department said in a statement, adding 37 people were still undergoing treatment. The victims included a three-year-old child and nine adults. Nomantu Nkomo-Ralehoko, the provincial head for health, said additional staff, including medics and nurses, were being mobilized to deal with the outbreak, which was centered in Hammanskraal, an area north of Pretoria. “We would like to reiterate and urge the public to avoid known or suspected contaminated food, water and surfaces and wash hands thoroughly with soap before handling food or after using ...
- Source: drugdu
- 115
- May 23, 2023
First diagnostic test for long COVID is available to patients across Australia

The first diagnostic test for long COVID is now available to patients across Australia. The test can help physicians diagnose long COVID by differentiating it from other diseases with similar symptoms, and to design personalized treatment approaches. Persistent COVID, or long COVID, is a multiorgan symptomatic complex with symptoms persisting over time. It affects patients who have suffered from acute COVID-19 infection. It is estimated by the US Centers for Disease Control that about 20% – 30% of patients who have suffered from COVID-19 may develop long COVID. The typical symptoms of long COVID, such as fatigue, brain fog, shortness of breath, insomnia, and a wide range of cardiovascular issues, are certainly not unique to one condition. A diagnostic test to identify patients with long COVID, using objective measures of immune biomarkers, is an essential first step for treatment. The simple blood-based test was developed by diagnostic testing company IGeneX ...
- Source: drugdu
- 110
- May 23, 2023
Study finds significant amount of clotting in the arteries of patients with STEMI and COVID-19

The latest analysis from The North American COVID-19 STEMI (NACMI) was presented today as late-breaking clinical research at the Society for Cardiovascular Angiography & Interventions (SCAI) 2023 Scientific Sessions. The findings show patients with an ST-elevated myocardial infarction, or STEMI, and COVID-19 had a significant amount of clotting in their arteries both before and after intervention. Importantly, clots were seen in multiple arteries in close to 30% of patients, a phenomenon observed in less than 5% of patients with heart attacks who do not have COVID-19. In the United States, someone experiences a heart attack every 40 seconds (CDC). Of these patients, more than 25% will experience a more severe type of heart attack, an ST-elevated myocardial infarction, or STEMI caused by the sudden, total blockage of a coronary artery. Pre-COVID-19 mortality in STEMI patients was below 5% (JACC). Previous NACMI research has shown that mortality jumps to 20% to ...
- Source: drugdu
- 207
- May 22, 2023
Novel gene-editing strategy leverages unusual genetic alteration to block HIV spread in cells

Genetic alterations that give rise to a rare, fatal disorder known as MOGS-CDG paradoxically also protect cells against infection by viruses. Now, scientists at the Lewis Katz School of Medicine at Temple University have harnessed this unusual protective ability in a novel gene-editing strategy aimed at eliminating HIV-1 infection with no adverse effects on cell mortality. The new approach, described online April 28 in the journal Molecular Therapy—Nucleic Acids, is based on a combination of two gene-editing constructs, one that targets HIV-1 DNA and one that targets a gene called MOGS—defects in which cause MOGS-CDG. In cells from persons infected with HIV-1, the Temple researchers show that disrupting the virus’s DNA while also deliberately altering MOGS blocks the production of infectious HIV-1 particles. The discovery opens up new avenues in the development of a cure for HIV/AIDS. Proper MOGS function is essential for glycosylation, a process by which some cellular ...
- Source: drugdu
- 108
- May 22, 2023

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