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A Dilemma for Governments: How to Pay for Million-Dollar Therapies

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A Dilemma for Governments: How to Pay for Million-Dollar Therapies

Whereas there are greater than six million individuals worldwide with sickle cell, most of whom dwelling in sub-Saharan Africa, the preliminary launches are anticipated to concentrate on tens of 1000’s of sufferers in the USA and Europe.

Document-setting worth tags for gene therapies have largely escaped the criticism that has adopted different trade pricing selections. The sentiment displays simply how highly effective most of the gene therapies are — medical doctors typically go so far as to name them cures — and their distinctive place as one-time therapies. Such a remedy has only one probability to earn cash, and in some circumstances can substitute persistent therapies that may in any other case be given for the remainder of a affected person’s life at a a lot larger cumulative value.

Nonetheless, for middle-income international locations, “if advantages of those therapies are rapid by way of well being however the potential financial savings occur sooner or later, that math might not work for them,” mentioned Rena Conti, a well being economist on the Questrom Faculty of Enterprise at Boston College.

Tay Salimullah, a Novartis govt, mentioned the corporate works intently with governments and well being plans contemplating whether or not to cowl Zolgensma, in some circumstances permitting them to unfold out their funds over time, like a mortgage, or providing a worth lower if the remedy doesn’t work.

In Brazil, the settlement with Novartis requires the federal government to separate funds for every remedy into 5 equal elements over 4 years. If a affected person dies, have to be completely ventilated or is unable to keep up sure motor capabilities two years after receiving Zolgensma, the federal government is not going to be required to make the following funds.

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Till six years in the past, there have been no authorized therapies for S.M.A., which impacts about one in 10,000 newborns. Infants with probably the most extreme type of the dysfunction had been despatched residence and their households had been informed to arrange for them to die.

Zolgensma and two different medication authorized since 2016 have opened up as soon as unimaginable prospects for S.M.A. sufferers. “I’m telling dad and mom to maintain placing cash of their faculty fund as a result of this child has a future,” mentioned Dr. Thomas Crawford, who treats S.M.A. sufferers at Johns Hopkins Medication.

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Why Do Dogs Put Their Paw on You? Canine Experts Explain What Your Pup May be Communicating

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Why Do Dogs Put Their Paw on You? Canine Experts Explain What Your Pup May be Communicating



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'Forever chemicals' found in US drinking water, map shows 'hot spots' of highest levels

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'Forever chemicals' found in US drinking water, map shows 'hot spots' of highest levels

The risk of having potentially harmful chemicals in your drinking water may depend on your zip code.

A study published in the journal Nature Geoscience on April 8 found that higher amounts of PFAS (perfluoroalkyl substances) were found in drinking water in certain parts of the U.S.

PFAS — also known as “forever chemicals” due to how slowly they break down — are a group of chemicals used during industrial processes and the manufacturing of consumer products. 

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Two of the main chemicals are perfluorooctanoic acid (PFOA) and perfluorooctane sulfonate (PFOS).

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There are many ways people can be exposed to these chemicals — including in drinking water.

A study published in the journal Nature Geoscience on April 8 found that higher amounts of PFAS (perfluoroalkyl substances) were found in drinking water in certain parts of the U.S. (Nature Geoscience)

To determine the prevalence of PFAS in the environment, researchers from the University of New South Wales (UNSW) in Sydney analyzed a global dataset of 273 studies dating back to 2004.

The studies included data for over 12,000 samples of surface water (water that collects on the ground) and more than 33,900 samples of groundwater (water found underground, below the surface).

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“We looked everywhere for PFAS concentrations in water data, including scientific journals and governmental reports and websites,” Denis O’Carroll, a UNSW engineering professor and senior author of the study, told Fox News Digital.  

“We compared PFAS concentrations in these water samples to international regulations. We also compared the types of PFAS analyzed to what we found in consumer products.”

Water sample

The studies included data for over 12,000 samples of surface water (water that collects on the ground) and more than 33,900 samples of groundwater (water found underground, below the surface). (iStock)

Nearly 70% of the samples had levels of PFAS that exceeded Canada’s minimum safety standards (30 nanograms per liter), while 6% had more chemicals than the European Union’s standard (100 ng per liter), according to the study findings.

In the U.S., PFAS “hot spots” were concentrated in the Midwest, New England and the West Coast, according to a map illustrating the findings — although the chemicals were also detected in other areas across the country.

Globally, Australia, Europe and China showed high levels.

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“To date, nobody has looked at the global extent of PFAS in our waters and compared it to international drinking water standards,” noted O’Carroll. 

“Our study found that a substantial fraction of sampled waters exceeded PFAS drinking water guidance values, with the extent of exceedance depending on the jurisdiction and PFAS source.”

Health risks of PFAS

Dr. Mark Fischer, regional medical director of International SOS, a health and security risk mitigation company headquartered in London, pointed out that most Americans also have these chemicals in their blood. 

“Although the use of these chemicals has declined in recent years, they are difficult to break down, so they are still found in some food, water and consumer products, as well as within the soil and the environment,” Fischer, who was not involved in the UNSW study, told Fox News Digital.

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Woman at doctor

Health risks associated with PFAS include cancers, elevated liver enzymes, lower birth weight, heart issues and higher cholesterol. (iStock)

Most people in the U.S. have been exposed to these chemicals, most likely through contaminated food or their drinking water, according to the Centers for Disease Control and Prevention. 

Health risks associated with PFAS include cancers, elevated liver enzymes, lower birth weight and higher cholesterol, Fischer said. 

“PFAS contamination has been identified in drinking water in all 50 states.”

Other potential dangers include heart issues, plus immune and developmental damage, according to the Environmental Protection Agency (EPA).

“PFAS contamination has been identified in drinking water in all 50 states, according to the Environmental Working Group,” Fischer said. 

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“That said, the levels of these chemicals vary throughout states and cities.”

Study limitations

The study did have some limitations, the researchers acknowledged. 

“We have measured a much wider range of PFAS in consumer products than in our waters,” said O’Carroll. 

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“As such, we don’t really have a great idea of the range of PFAS in our waters.”

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The data was also limited to what the researchers could find, he noted.

“Just because we can cheaply use a chemical doesn’t mean we should.”

“While we had data for over 45,000 water samples, even more data would be useful, especially for parts of the world where we had limited data.”

The actual amount of PFAS in water could be higher than what the study results imply, O’Carroll added.

Happy beautiful young woman drinking water

Most people in the U.S. have been exposed to PFA chemicals, most likely through contaminated food or their drinking water, according to the Centers for Disease Control and Prevention.  (iStock)

“Current monitoring practices probably underestimate PFAS in the environment, given the limited suite of PFAS that are typically quantified but deemed of regulatory concern,” he said.

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These are just one of the many types of chemicals that are used in daily life, O’Carroll noted.

“As a society, we need to consider the chemicals we use and reduce our use of some,” he advised. 

“Just because we can cheaply use a chemical doesn’t mean we should.”

Latest PFAS regulations

On April 10, the EPA finalized new limits on the amount of PFAS permitted in drinking water.

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The new standards could reduce exposure for 100 million people, potentially preventing thousands of deaths and tens of thousands of serious illnesses, the agency said in a news release.

On April 10, the EPA finalized new limits on the amount of PFAS permitted in drinking water. (Pavlo Gonchar/SOPA Images/LightRocket via Getty Images)

This is the “first-ever national, legally enforceable drinking water standard” to protect people from the health risks of PFAS, the agency stated.

The 66,000 public drinking water systems in the U.S. will have three years to reduce PFAS levels to meet the new standards, according to the release.

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The EPA also announced $1 billion in new funding, provided through the Bipartisan Infrastructure Law, to enable PFAS testing and treatment of public water systems and private wells.

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Fox News Digital reached out to the EPA and the American Water Works Association for comment.

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Lack of sleep could be a factor in a 'silent epidemic,' experts warn

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Lack of sleep could be a factor in a 'silent epidemic,' experts warn

More than a third of adults fail to get the recommended seven to eight hours of sleep each night — and the scarcity of shuteye can have a surprising effect.

Lack of sleep can lead to what some experts refer to as a “silent epidemic” — a condition called non-alcoholic fatty liver disease (NAFLD), which affects more than one in four American adults, according to the American Heart Association.

What is non-alcoholic fatty liver disease (NAFLD)?

As its name implies, NAFLD is marked by the presence of fat in the liver.

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The primary cause is weight gain, along with metabolic risk factors such as type 2 diabetes, high blood pressure, high triglycerides and obstructive sleep apnea, according to Ibrahim Hanouneh, a gastroenterologist with MNGI Digestive Health in Minnesota.

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Lack of sleep can lead to what some experts refer to as a “silent epidemic” — a condition called non-alcoholic fatty liver disease (NAFLD), which affects more than one in four American adults. (iStock)

“Heavy alcohol consumption can also lead to fatty liver, but NAFL refers to fatty liver that occurs in the setting of metabolic syndrome and weight gain in the absence of heavy alcohol consumption — hence the name ‘non-alcoholic,’” Hanouneh told Fox News Digital.

The condition is also known as MASLD (metabolic dysfunction associated steatotic liver disease) to reflect the association between fatty liver and metabolic syndrome.

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A family history of fatty liver can also increase the risk, noted Hanouneh, who is also co-author of the book “Regenerative Health: Discover Your Metabolic Type and Renew Your Liver for Life.”

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NAFL is known as the “silent epidemic” because it often has no symptoms. 

“In some studies, fatty liver has affected 25% to 33% of the general population — almost one out of four individuals — but the vast majority of individuals have no symptoms at all, particularly in the early stages,” said Hanouneh.

NAFLD

The primary cause of NAFLD is weight gain, along with metabolic risk factors such as type 2 diabetes, high blood pressure, high triglycerides and obstructive sleep apnea. (iStock)

Some people may have non-specific symptoms, such as fatigue, foggy memory and abdominal pain.

“Generally speaking, fatty liver disease does not cause major symptoms until it is too late — for example, when the patient has already developed cirrhosis or liver cancer,” Hanouneh said.  

Fatty liver disease is associated with an increased risk of liver cirrhosis and liver cancer.  

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“The vast majority of individuals have no symptoms at all, particularly in the early stages.”

“NAFLD has become the leading indication of liver transplantation and the leading cause of liver cancer in the Western world,” noted Hanouneh.  

Patients with this disease are also at an increased risk of cardiovascular events, such as heart attacks and strokes, he warned.  

“Some studies also suggest that patients with NAFL are at an increased risk of getting type 2 diabetes,” he added.

Woman fatty liver

Patients with this disease are also at an increased risk of cardiovascular events, such as heart attacks and strokes, a doctor warned.   (iStock)

Not all people with NAFLD experience liver damage. Nonalcoholic steatohepatitis (NASH), the more severe form, leads to swelling or inflammation of the liver and damage to liver cells, according to the American Liver Foundation’s website.

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This can ultimately lead to fibrosis, or scarring, of the liver.

Link between sleep and NAFLD

Lack of high-quality sleep has been linked to weight gain, an increase in hunger and adverse blood sugar control in multiple studies, according to Kristin Kirkpatrick, a registered dietitian at Cleveland Clinic and co-author of “Regenerative Health.”

“Insulin resistance is a major risk factor for abnormal amounts of fat in the liver,” said Kirkpatrick.

PITTSBURGH BOY, 10, NEEDS SECOND LIVER TRANSPLANT TO SAVE HIS LIFE: ‘ONLY POSSIBLE THROUGH LOVE’

The risk is particularly high for postmenopausal women, she noted. One study found that chronic short sleep duration led to a 20% increase in insulin levels among that group, compared to 15% overall.

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“The stress on the body caused by a lack of sleep can cause adverse metabolic changes that ultimately may lead to NAFLD,” Kirkpatrick said.

Woman awake

Lack of high-quality sleep has been linked to weight gain, an increase in hunger and adverse blood sugar control, studies have shown. (iStock)

The expert also cited a new study in JAMA (the Journal of the American Medical Association) showing that short sleep duration increased the risk for type 2 diabetes.

“Though some risks could be offset by a healthy diet, a majority could not be reversed by a nutrient-rich dietary pattern,” she said.  

ALWAYS FEELING TIRED? EXPERTS SHARE 4 COMMON CAUSES OF DAYTIME FATIGUE

“Type 2 diabetics have a significant increase in risk of developing NAFLD.”

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Maintaining consistent sleeping hours may help with weight management, Kirkpatrick told Fox News Digital. 

“That means going to bed and waking up at the same time on weekdays and weekends.”

Screening, treatment and prevention

Due to the “silent” nature of NAFLD, experts stressed the importance of screening for fatty liver in patients with risk factors — even if there are no signs or symptoms of liver disease.

“NAFLD is a preventable disease,” Hanouneh told Fox News Digital. 

“It is also a reversible disease at early stages.”

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Liver scan

Imaging tests, such as ultrasounds or CT scans, can visually assess the appearance of the liver. (iStock)

Three main types of tests are used to diagnose the condition, according to JAMA Network.

Blood tests can measure inflammation in the liver. 

Imaging tests, such as ultrasounds or CT scans, can visually assess the appearance of the organ.

“NAFLD is a preventable disease — and reversible at early stages.”

There are also some newer tests that measure the amount of fat in the liver — “such as transient elastography, an ultrasound-based test that measures how stiff the liver is” — but they may not be very reliable for diagnosing more advanced liver disease, JAMA Network stated.

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For those who have been diagnosed, a low-carbohydrate diet is key to managing fatty liver disease, experts agreed.

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“Studies suggest that a low-carb diet that leads to weight loss of 5% to 10% can potentially reverse fatty liver disease,” Hanouneh said.  

“Also, cut back or abstain from alcohol depending on the degree of fatty liver disease.”

Man with fatty liver

It’s important to follow up with a health care professional to manage metabolic risk factors, doctors advised. (iStock)

It’s also important to follow up with a health care professional to manage metabolic risk factors, he said — particularly type 2 diabetes, which is “crucial in the management of fatty liver disease.”

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For people with NAFLD, Hanouneh also recommended incorporating regular exercise, particularly interval training, three times a week.  

Coffee has also shown potential benefits for fatty liver.  

“I typically recommend one to two cups of black coffee a day if possible,” Hanouneh said.

For more Health articles, visit foxnews.com/health

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