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US drug firm offers cure for blindness – at $425,000 an eye

Spark Therapeutics says ‘responsible price’ for Luxturna gene therapy ensures access for patients with retinal defect

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Powered by Guardian.co.ukThis article titled “US drug firm offers cure for blindness – at 5,000 an eye” was written by Rob Davies, for The Guardian on Wednesday 3rd January 2018 18.15 UTC

A drug whose inventors claim it can cure a rare form of blindness is to be one of the most expensive medicines ever sold at 0,000 (£630,000).

Luxturna is injected directly into the eye to address the root cause of visual impairment by replacing a defective gene in the retina. It is the first gene therapy to be approved for use in the US, and was given the go-ahead by the Federal Drug Administration last month. However, the price of the treatment has only just been revealed.

Spark Therapeutics, the company behind the treatment, had previously claimed the treatment was worth m, citing the cost of a lifetime of blindness in lost earnings and wages for caregivers. But the firm said it had settled for the lower price of 0,000, or 5,000 per eye. It will also offer ways to spread the cost to health insurers, which have expressed concern about their ability to cover the expense.

“We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients,” said Spark Therapeutics’ chief executive, Jeff Marrazzo.

Luxturna is one of an emerging breed of gene therapies that differ from more established medicines administered over a period of time. Such treatments involve a one-off procedure to alter defective DNA, allowing the body to repair the problem itself.

They include a treatment for haemophilia and another for so-called “bubble baby” syndrome, where sufferers have to live in a sterile environment, which is to be offered on the NHS despite a £500,000 price tag.

The sky-high price of some drugs – and so-called price-gouging by drug firms – became an issue in the US presidential election after Martin Shkreli, a US hedge fund entrepreneur, bought the drug Daraprim, used in the treatment of Aids and cancer, and hiked its price from .50 to 0.

But the starting price of some new drugs has soared. Glybera, a gene therapy for a rare protein disorder, was launched in 2012 with a price tag of .2m. However, it was never approved in the US and was discontinued by manufacturers uniQure because of a lack of demand.

a vial of Luxturna (voretigene neparvovec-rzyl)
A vial of Spark Therapeutics’ Luxturna (voretigene neparvovec-rzyl), which was approved for use by the FDA on Tuesday. Photograph: AP

Gene therapy is not alone in commanding staggering sums, particularly when it comes to treatments for rare diseases. Soliris, a drug that treats a condition called paroxysmal nocturnal hemoglobinuria that attacks red blood cells, can cost up to 0,000 a year, while Elaprase, used in the treatment of Hunter syndrome, costs 0,000 a year.

Luxturna could potentially become available free on the NHS in the UK after being submitted for approval by the European Medicines Agency (EMA). A green light from the EMA is a prerequisite for approval by the National Institute for Health and Care Excellence, the UK’s healthcare costs watchdog.

In clinical trials, injections of Luxturna restored eyesight to people with severe visual impairment due to retinal dystrophy. Spark estimates that up to 2,000 people in the US suffer from the condition, with the number of sufferers rising to 6,000 when Europe and other markets where it could sell the treatment are included.

The company said it had agreed bespoke deals with US insurers, which cover the cost of most US prescriptions, and they will get a refund if the drug doesn’t work as expected.

Spark is also discussing a proposalfor insurers to pay for the drug in instalments over several years. The Philadelphia-based company said it would also pay transport costs not covered by insurance to help patients get access to treatment centres.

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Prezzo in Salisbury cordoned off by police after man and woman fall ill

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Police have sealed off a restaurant in Salisbury and the surrounding area after two people were taken ill.


The ambulance service called officers to Prezzo, in High Street, at 18:45 BST following “a medical incident” involving a man and a woman.


A Wiltshire Police statement said it had cordoned off the area as a precaution while it established “what has led them to fall ill”.
A witness reported seeing a person in a a hazardous material suit attend.

(BBC)

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California to launch its ‘own damn satellite’

California is set to launch a satellite to track greenhouse gases, as former US Secretary of State John Kerry and island nation leaders warned that the world is far off course to avoid the worst effects of rising temperatures.

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Powered by Guardian.co.ukThis article titled “California to launch its ‘own damn satellite’ to track greenhouse gases” was written by Emily Holden and Oliver Milman in San Francisco, for theguardian.com on Friday 14th September 2018 20.49 UTC

California is set to launch a satellite to track greenhouse gases, as former US Secretary of State John Kerry and island nation leaders warned that the world is far off course to avoid the worst effects of rising temperatures.

Gov. Jerry Brown announced plans for the satellite on the last day of a climate change summit hosted by San Francisco, in a final rebuke to President Donald Trump’s denial of man-made warming.

“With science still under attack,” Brown said “we’re going to launch our own satellite, our own damn satellite, to figure out where the pollution is.” Brown said the satellite will help pinpoint the source of planet-warming emissions.

California will team up with Planet Labs, a company run by ex-Nasa scientists. The data collected, including on carbon dioxide emissions and methane leaks from oil and gas operations, could be made public as part of a partnership with the advocacy group Environmental Defense Fund. The new project comes as Trump has proposed slashing Nasa climate research mission budgets. It is one of dozens of commitments of mixed significance unveiled by states, cities and businesses at the event.

Despite the optimism on show at the summit, Kerry said climate efforts must ramp up.

“I am going to tell the truth, and the truth is we are not anywhere near where we need to be with respect to the overall challenge of climate change,” said Kerry, who worked to secure the 2015 global Paris climate agreement under former president Barack Obama.

Kerry blasted Donald Trump for deciding to leave that deal, calling it “one of the single greatest acts of irresponsibility by a president of the United States anywhere at any time.”

Leaders of the countries already suffering most from sea-level rise and ocean acidification echoed Kerry’s concerns, saying that international action is slowing.

“The world has lost, all of us have lost, momentum since Paris in 2015. Although the rate of increase has slowed, we’ve not yet peaked our global emissions. But we must do so by 2020. We really cannot afford to wait any longer,” said Mia Mottley, prime minister of the Caribbean island nation of Barbados.

Mottley’s country is in the direct path of hurricanes that are growing in strength and may narrowly avoid a more direct hit from tropical storm Isaac this week.

The world is set to watch temperatures rise 3C above pre-industrial levels by the time a child born today is old, Mottley said, even if countries adhere to the goals they said.

Frank Bainimarama, prime minister of Fiji, said countries need to speed their work.

“We all know that the levels of ambition in our national plans need to be ramped up because we are not on track to meet the targets of the Paris agreement,” Bainimarama said.

Former US vice-president Al Gore struck a more positive tone.

“We must do it. We can do it. I’m convinced ever more because of the success of this summit here in San Francisco that we will do it,” he said, reminding that the US has not technically left the Paris deal yet and that a new president could re-enter.

The warnings were at odds with the overall atmosphere of the summit.

On the eve of the gathering, California governor Jerry Brown signed a bill that would make the state’s electricity supply carbon-free by 2045. A separate executive order by Brown is more sweeping, committing to net zero emissions across the entire California economy, also by 2045.

Other cities and regions from around the world have followed this with various pledges, with New York City promising $4bn to renewable energy and clean water and cities including Los Angeles, Tokyo, Honolulu, Oslo and Greater Manchester pledging to build energy efficient buildings or deploy fleets of electric buses.

A group of 29 philanthropists committed $4bn over five years to combat climate change, the largest such investment of its kind, while companies such as Ikea, Walmart and Unilever promised to reduce emissions through measures such as electrified trucks for deliveries and action to prevent deforestation in the tropics.

Jonathan Pershing, the State Department’s climate negotiator under Obama, said the summit brings hope to the climate cause.

“The story here is optimistic. The question here is does the optimism translate, and can this message get out globally,” Pershing said. “There is a good broad cross-section of people from around the world, but it’s just a few thousand people, and it’s a problem that’s going to require engagement by millions.”

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Christine Blasey accuses Kavanaugh of assault in letter to senator

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Update:Christine Blasey Ford, the woman who wrote the letter accusing Supreme Court nominee Brett Kavanaugh of sexual assault, is going public with her story, saying she thought he might kill her. More to come.

‘I thought he might inadvertently kill me,’ said Ford, now a 51-year-old research psychologist in northern California, to The Washington Post. ‘He was trying to attack me and remove my clothing.’

A woman is accusing Supreme Court nominee Brett Kavanaugh of assaulting her when they were in high school in the early 1980s, according to a source familiar with the allegations, which were relayed in a letter to Sen. Dianne Feinstein earlier this summer.

CNN reports the letter details an incident when the woman, who has not come forward publicly, attended a party with Kavanaugh and others in a suburban Maryland home. Feinstein, the top Democrat on the Senate Judiciary Committee, has referred the letter to the FBI.

Kavanaugh physically pushed her into a bedroom, the accuser said. Along with another male, Kavanaugh locked the door from the inside and played loud music that the accuser said precluded successful attempts to yell for help.

Both men were drunk, she said, and Kavanaugh attempted to remove her clothes.

At one point, Kavanaugh was on top of her laughing as the other male in the room periodically jumped onto Kavanaugh.

Kavanaugh held his hand over her mouth at one point, and she said she felt her life was inadvertently in danger.

She said she was able to leave the room and go into a hallway bathroom. After Kavanaugh and the other male began talking to others in the house, she went home.

There is no indication the woman reported the incident to law enforcement at the time, but she said she has received medical treatment regarding the alleged assault. The woman also declined to come forward publicly after sending the letter to Feinstein. The accuser’s name was redacted before Feinstein forwarded it to the FBI.

In a statement Friday, Kavanaugh denied the allegation.

“I categorically and unequivocally deny this allegation. I did not do this back in high school or at any time,” he said.

Kavanaugh testified for three days before the Senate Judiciary Committee last week, where the issue was not raised. The Judiciary panel is scheduled to consider Kavanaugh’s nomination next Thursday, and the full Senate may vote on confirmation later this month.

The New Yorker first reported the details of the letter to Feinstein. The woman declined a request from the magazine for comment.

Old Article:

Sen. Dianne Feinstein (D-CA) has said that she possesses a sensitive document about Supreme Court nominee Brett Kavanaugh and is referring the matter to the Justice Department.

In a statement she said:

“I have received information from an individual concerning the nomination of Brett Kavanaugh to the Supreme Court,” Feinstein said in a statement. “That individual strongly requested confidentiality, declined to come forward or press the matter further, and I have honored that decision. I have, however, referred the matter to federal investigative authorities.”

The document in question is believed to be a letter detailing an interaction between an unnamed woman and Kavanaugh dating back to their time together in high school. 

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