Luxturna gene therapy approved for vision loss

A research scientist works in Spark Therapeutics’ laboratory.				Spark Therapeutics Inc

This rare genetic defect causes severe visual impairment beginning in infancy and can eventually lead to blindness. The manufacturers say the price is a bargain.

Luxturna is still significantly more expensive than almost every other medicine on the global market, including two other gene therapies approved earlier a year ago in the U.S. Approved last month, Luxturna, is the nation's first gene therapy for an inherited disease. It is unclear how long the benefit of the treatment will ultimately last.

Besides Luxturna, several other medications are near the million-dollar price mark, although they are often administered in several doses.

"But it's the right move", he continues, "and highlights a recent trend toward (relative) pricing moderation with big implications for the drug industry".

Spark Therapeutics gained approval from the Food and Drug Administration in December for Luxturna (voretigene neparvovec), ahead of its February user fee action date.

The announcement comes amid a growing debate in the United States over the high cost of medicine, particularly for supposedly innovative treatments.

"We wanted to balance the value and the affordability concerns with a responsible price that would ensure access to patients", said Jeffrey Marrazzo in an interview Wednesday. But gene therapies are given only once, with potentially permanent effects.

The next year will be critical in showing that gene therapies are marketable.

East Africa anticipates benefits from Kenya - US direct flights
Kenya Airways will be using its vast African connecting routes to pull passengers to fly direct to the US. The airline already serves Africa, Europe, Middle-East, Indian sub-continent and Asia.

Investors bet on pickups and SUVs despite low United States sales
Fiat Chrysler's sales fell 11 percent, while Toyota's sales dropped 8 percent and Nissan's sales were down 9.5 percent. Despite the usual year-end push to get vehicles out the door, most automakers endured tough sledding in December.

President Of The Mormon Church Dies
That said, after a notable person's death, it's important not to sugarcoat their legacy by ignoring the terrible things they did. And, before I knew it, I found myself eating a cookie while standing face-to-face with him in a corner of the room.

Spark Therapeutics has devised a set of payment and access programs to make Luxturna more accessible while removing additional costs and risks that may face insurers and hospitals, CNBC News said.

As questions swirl around the cost and effectiveness of gene therapy, it could not be doubted that Luxturna is a breakthrough in the development of this kind of medicine.

But even compared to other one-time gene therapies Luxturna is still an outlier. "Our system can not handle unjustified prices like this, and the new payment models announced today are merely a way to disguise a price that is simply too high", said David Mitchell, a patient with incurable blood cancer and the president of Patients For Affordable Drugs. And the company is also working on covering patients' out of pocket costs as part of the overall price.

Kymriah is a leukemia treatment made by pharmaceutical giant Novartis.

The FDA then approved Luxturna a couple of weeks ago.

Fundus albipunctatus, which makes seeing in low light hard and is sometimes called 'night blindness'.

Spark Therapeutics CEO Jeff Marrazzo co-authored a recent blog post in the journal Health Affairs that said "the current payment system - accustomed to ongoing treatment of chronic diseases - creates significant challenges" for gene therapies and other treatments.

"Although much of this may still sound like the realm of mad scientists tinkering with the human body, gene therapy is an accepted experimental technique that is now being used to help patients with certain types of cancer to "target" specific antibodies that can be used to fight the disease", according to a blog post by Science Care, which runs a whole body donation program.

Related News:

  • Norv Turner agrees to become Panthers OC

    Norv Turner agrees to become Panthers OC

    Rivera spent four years under Turner at San Diego from 2007 to 2010, including the last three years as the defensive coordinator. The team is interviewing Norv Turner for its vacant offensive coordinator position, said a person familiar with the situation.
    Arizona trails Oregon State 22-21 at halftime

    Arizona trails Oregon State 22-21 at halftime

    Pritchard put the game on ice with two free throws in the closing moments that put the game out of reach for the Sun Devils. The Ducks face a tough road test at Arizona State and Arizona before returning home to meet USC and UCLA .
    Mira Sorvino Hopeful

    Mira Sorvino Hopeful "Time's Up" Stays Focused on Harassment

    In a piece for the Huffington Post , Sorvino explained that she believes Dylan and stands by her "gratitude and solidarity". The actress, who apologized for taking so long to address working with Allen , also pledged to never work with him again.
  • Cameron Jordan Trolls Cam Newton By Sending QB Wine After Saints' Win

    Cameron Jordan Trolls Cam Newton By Sending QB Wine After Saints' Win

    Brees, who turns 39 next week, will be hoping to lead his team to a better result than they managed in week 1. "We beat Minnesota. He was checked for a possible concussion because of how it looked, but that wasn't what was going on. "He did not.
    NFL Playoffs: Jaguars End Bills' Cinderella Story With 10-3 Victory

    NFL Playoffs: Jaguars End Bills' Cinderella Story With 10-3 Victory

    Ben Koyack caught the game-winning touchdown from Blake Bortles on fourth and goal (Tommy Gilligan/USA Today) More Buffalo magic? The defense overall was outstanding - they reacted well when Taylor broke contain and really didn't allow many busts in coverage.
    Oil tanker Sanchi partially explodes in East China Sea

    Oil tanker Sanchi partially explodes in East China Sea

    Rescuers from China, South Korea, and Japan have been struggling to control the blaze and find survivors, if any. That warning of a potential environmental disaster has come for South Korea's Ministry of Oceans and Fisheries.
  • Turkey summons U.S.  charge d'affaires over Syria: Turkish sources

    Turkey summons U.S. charge d'affaires over Syria: Turkish sources

    Turkey entered the war in Syria on the U.S.'s side, but has grown increasingly distant over US support for Kurdish militants. Turkish President Tayyip Erdogan speaks during a meeting at the Presidential Palace in Ankara, Turkey, January 10, 2018.
    Cuba Rejects Allegations by High-Ranking US Official

    Cuba Rejects Allegations by High-Ranking US Official

    Charles Rosenfarb , the State Department's chief doctor, also dismissed speculation that the illnesses were psychosomatic. The State Department continues to be deeply concerned about the safety and security of our personnel.
    Jon Gruden Publicly Confirms He'll Join Raiders If Offered Job

    Jon Gruden Publicly Confirms He'll Join Raiders If Offered Job

    Del Rio and Pagano are likely to be joined by several other sacked head coaches over the next couple of days. Morton was a practice squad players with the Raiders throughout the mid 1990s.
  • ASUS Republic of Gamers unveils latest gaming lineup at CES 2018

    ASUS Republic of Gamers unveils latest gaming lineup at CES 2018

    The backlit keys are individually programmable and there is a swappable, illuminated acrylic insert emblazoned with the ROG badge. Next up is the Asus Bezel-free Kit, which is made of vertical refractive lenses meant to hide the bezels in multi-monitor setups.
    Eden Hazard voted into UEFA Fan's Team of the Year

    Eden Hazard voted into UEFA Fan's Team of the Year

    Marcelo, Sergio Ramos, Toni Kroos and Luka Modric join Ronaldo in the team, while Messi is the only Barcelona player to feature. The team also included three players from the Juventus team who lost the Champions League final to Real Madrid .
    Messi equals Muller record as Barcelona beat Levante

    Messi equals Muller record as Barcelona beat Levante

    Paulinho added the gloss to the scoreline late on, converting from close range after Messi's fine dribble and cross. "There is an agreement that Lionel will remain at the club as long as they are playing in a top league".


Most liked

Why IKEA wants women to pee on their new ad
The advert is now in IKEA Sweden's women and lifestyle magazine, Amelia , but as of yet has not appeared in the UK. The pregnancy test strip uses antibodies that bind to the pregnancy hormone hCG, which causes it to change color.

OR drivers concerned about self-service gas stations following new law
New Jersey has had a ban on self-pumping just as long as we have and you don't see everyone calling them a bunch of bumpkins. Kyle Allen spoke of the true horror of having to fill up himself: "One time, my dad came to OR and pumped his own gas".

MINI hatchback and convertible get a refresh, but you probably can't tell
The original also gets three new paint color options: Emerald Grey metallic, Starlight Blue metallic, and Solaris Orange metallic. All of the new models can also be customized via the new Mini Yours Customised program we talked about before.

North Korea missile makers hit with USA sanctions
The country will "deal with the U.S.'s most ferocious declaration of war with fire surely and definitely", the report said. Industry sources say China still supplies about 3.8 million barrels of crude a year to North Korea via an aging pipeline.

Record 656 patients waiting for hospital bed
Across the border in Portlaoise Hospital, 25 patients are awaiting a bed, while there are 31 in a similar situation in Mullingar. The HSE, meanwhile, is urging high-risk groups to get the flu vaccine as the flu has started actively circulating in Ireland.

I-65 south remains closed near Seymour due to fiery crash
Westbound traffic is being diverted off at Grant Street and eastbound traffic is being diverted off at Cline Avenue, police said. After the burned out damage was cleared away, IN DOT crews were able to assess the damage to the roads.

1 officer dead in Colo. 'ambush'
Monday for a Colorado sheriff's deputy who was gunned down after responding to a disturbance call in suburban Denver early Sunday. Officer Parish's body was escorted from hospital by a procession of dozens of law enforcement vehicles late on Sunday morning.

Zuma to set up commission to probe influence-peddling
At 4.30pm, the rand was up 0.28% to R12.35 against the dollar from a close of R12.38 overnight in NY . But talks of a motion to remove Zuma being forced into the agenda have been gaining momentum.

Red Fort attack accused Bilal Kawa arrested after 18 years
However, the family of Kawa has rejected Intelligence's claims that he was involved in the Red Fort terror attack . Earlier, terrorist Mohammad Arif alias Ashfaq, who was convicted in the case, had been awarded death sentence.

E-cigarettes may be a gateway to smoking for teens, researchers say
The research also showed that teens who used more than one tobacco product were more likely to move on to traditional cigarettes. Polyuse of two or more noncigarette tobacco products was associated with greater odds of cigarette smoking.

Canadian fund to buy Westinghouse
Limited Partnership Units now has a consensus rating of "Buy" and a consensus price target of $29.66. Four research analysts have rated the stock with a buy rating, Brookfield Business Partners L.P.

Liverpool January target likely to stay put over World Cup fears
That is because Jurgen Klopp's side, as well as Manchester United , are in the mix for the Chilean now. "He's young. However, Jardim added: "If I stayed here for 10 years, I'd want Lemar to stay with me for 10 years".

Enzo Amore Predicts Ronda Rousey set for 'Rude Awakening' in WWE
Besides, Rousey is unlikely to work a full-time schedule in WWE, irrespective of when she debuts. Levesque was in Los Angeles to speak on a Television Critics Association panel.

Controversial US Senate candidate Joe Arpaio questions Obama's birth certificate
Arpaio refused to go into the details but he believed he had enough evidence to support his claims , CNN reported . The former six-term sheriff of metro Phoenix also said he and Trump have both been persecuted by the U.S.

Macron appeals to China for partnership on climate
He also urged European nations to take a more "coordinated approach" on trade with China . A position he did not hide during the visit .