Vt. Mom Begs FDA: Save My Other Son

Jenn McNary, a mother of six from Saxtons River, Vt., is desperate.

Both her boys have Duchenne muscular dystrophy, but only her 10-year-old Max has access to a wonder drug that appears to be reversing the symptoms of this deadly disease.

His 13-year-old brother Austin is languishing in a wheelchair while Max has been able to take the drug eteplirsen through a highly successful clinical trial.

After 60 weeks on an IV infusion, Max was able to participate in a three-mile Halloween walk.

“It’s the first time ever — he’s never been able to walk that far. He’s always gone with a wheelchair, even as a toddler,” said McNary, 32. “He actually doesn’t look like a Duchenne kid at all. And his balance is great.”

“People all over the world are calling it a miracle,” she said of the drug.

Now, McNary has written petitioned the Food and Drug Administration to give accelerated approval of the medication, the fastest way to help Austin and other boys with the disease.

Both boys, whose last name is Leclair, have the same gene mutation that the drug targets and will eventually kill them. Austin was diagnosed at 3 and Max at 3 months.

McNary and her husband Craig are also raising four other healthy children in a second marriage.

There is no cure for Duchenne muscular dystrophy. Until now, doctors have only been able to use steroids, which just temporarily delay the inevitable loss of muscle strength.

“My brother says he’s doing it for me, that he’s trying really hard,” Austin told ABCNews.com in August. “That’s why he wanted to do it.”

Austin was not allowed to participate in the clinical trial because one of the inclusion criteria was that he be able to complete a six-minute walk.

“This has been a bitter-sweet journey for us,” McNary wrote in a letter to the FDA this week. “As we watch Max get better, we also watch his older brother, Austin, 13, get worse. He suffers, silently, as his disease progresses.”

Duchenne muscular dystrophy affects one in 3,500 male births, about 20,000 children in the United States and 300,000 worldwide, according to Cure Duchenne, one of three organizations that have funded the clinical trial.

The muscular disease strikes between the ages of 3 and 5 as boys progressively lose their ability to walk. Eventually, they are wheelchair bound, their upper body strength fails, and, like Austin, they eventually cannot raise their arms to feed themselves.

Later, their breathing is affected and they require tracheotomies and breathing assistance. Eventually, the heart and lungs fail.

Parents of children who were in the clinical trial of eteplirsen at Nationwide Children’s Hospital in Columbus are calling it a “wonder drug.”

According to McNary, all 12 children in the double-blind study received “some benefit” from the drug. It has no known side effects.

“Even two boys who stopped walking before taking it have stronger upper bodies and their hearts are strong,” she said. “They have progressed to stable.”

Muscular Dystrophy Drug Could Stabilize Disease

If this exon-skipping drug is approved, she estimates 15 percent of boys with Duchenne could be helped, those with the type that skips exon 51. As a class of drugs, they could up to 85 percent of boys with the disease.

Stock prices for its manufacturer, Sarepta Therapeutics. , have soared.

If Sarepta Therapeutics can get accelerated approval, the drug could be available in six to nine months, according to McNary. Otherwise, the wait could be four or five years — too late for Austin.

“We are very encouraged by the data we have seen to date,” said Chris Garabedian, president and CEO of Sarepta Therapeutics, which makes the drug and is pressing the FDA to take action.

“If we start using the drug earlier in patients, we might be able to stabilize whatever state they are in for a longer period of time,” said Garabedian. “We are not going to end up creating Olympic athletes from this drug, but we are encouraged this could really halt or slow the progression.”

McNary is reaching out to media and online petition sites to encourage as many people as possible to write letters of support to the FDA.

But as she waits approval, Austin gets weaker. In the last few months, he has lost all upper body control and must be lifted 100 percent of the time.

Just recently, he was diagnosed with sleep apnea and must go on a nighttime machine to keep his lungs inflated.

Austin keeps his spirits high, according to McNary.

On Halloween, he dressed his wheelchair up as a hot dog stand, carrying his dachshund in a cloth bun. And just recently, his father and uncle took him hunting. They held up the gun for Austin and he shot his first buck — an eight-pointer.

McNary is convinced that if the FDA can move on approving the drug that has healed Max, it can also help Austin.

Until then, he’s “hanging in there,” she said. “He has a huge zest for life.”

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Clarke’s 218 puts Australia on front foot

BRISBANE (Reuters) – Australia captain Michael Clarke scored a brilliant unbeaten double century to give the hosts a remarkable 37-run first innings lead on the fourth day of the first test against South Africa on Monday.

Supported first by a maiden century from opener Ed Cowan in a record stand of 259, and then by Mike Hussey‘s 86 not out, Clarke’s 218 helped lift Australia from 40 for three when he took to the crease on Sunday to 487 for four when stumps were drawn.

It was Clarke’s sixth test century, and his third double hundred, in the 15 tests since he was named captain last year in the wake of the Ashes humiliation and Australia’s quarter-final exit at the World Cup.

Although by no means a chanceless knock, the 31-year-old played with patience when South Africa’s vaunted pacemen got anything out of the Gabba track before punishing anything loose with some fine shot-making.

When he carried his bat back to the pavilion at the end of the day to the raucous cheers of a sparse crowd at the famous Brisbane ground, Clarke had faced 350 balls over 504 minutes and scored 21 fours.

“I’m very happy with that,” Clarke, who accumulated his 1,000 test run of the year during the innings, said in an interview on the boundary.

“I didn’t feel great at the start and I think Ed Cowan batted beautifully.

“We’re in a great position with a 30-odd lead. I’d like another 70 odd runs in the morning and then I want to have a crack with the ball. We’ll see what happens.”

Cowan departed for 136 in heartbreaking fashion just before tea, run out at the non-striker’s end when Dale Steyn got a finger to a Clarke drive that hit the stumps and the opener was caught out of his crease backing up.

RECORD PARTNERSHIP

His partnership with Clarke was an Australian record for the fourth wicket at the Gabba, beating the 245 Clarke and Mike Hussey made against Sri Lanka in 2007.

Cowan’s wicket was the only wicket to fall on the day and Hussey started pouring on the runs as if determined to get the record back for his own partnership with his captain.

The 37-year-old bucked his poor recent form against South Africa by reaching his half century off just 68 balls with a drive through long-off and was closing on a century of his own when play ended.

It was Hussey’s cut four off Morne Morkel with which Australia overhauled South Africa’s first innings tally of 450 and put themselves in with an unlikely chance of even winning a test which lost an entire day to rain on Saturday.

Clarke’s negotiation of the “nervous nineties” for his century had been fraught and he was nearly run out going for a second run that would have brought him to the hundred mark.

There were no such jitters on his approach to the two hundred mark, which he passed by slapping the ball through mid-on for two runs before giving the badge on his helmet another kiss.

Cowan’s century was a retort to those critics who have consistently questioned his place in the team since he made his debut in last year’s Melbourne test against India.

The 30-year-old lefthander reached the mark two overs after lunch by pulling a short Vernon Philander delivery for four to the square leg boundary, beginning his joyous celebrations before the ball hit the rope.

South Africa’s number one test ranking is on the line in the series, which continues with matches in Adelaide and Perth after Brisbane.

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Microsoft’s Surface tablet has “modest” start: Ballmer

PARIS (Reuters) – Microsoft Corp‘s new Surface tablet – its challenger to Apple‘s iPad – had a “modest” start to sales because of limited availability, Microsoft Chief Executive Steve Ballmer told French daily Le Parisien.

The world’s largest software company put the Surface tablet center stage at its Windows 8 launch event last month in its fightback against Apple and Google in the exploding mobile computing market.

“We’ve had a modest start because Surface is only available on our online retail sites and a few Microsoft stores in the United States,” Ballmer was quoted as saying.

Meanwhile, 4 million upgrades to Windows 8 were sold in the three days following the system’s launch, Ballmer added. (Reporting by Lionel Laurent; Editing by David Cowell)

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Petraeus' aide wondered about Broadwell's behavior, qualifications

Retired Gen. David Petraeus, who resigned as CIA director last week after admitting an extramarital relationship, could possibly face military prosecution for adultery if officials turn up any evidence to counter his apparent claims that the affair began after he left the military.

The affair between Petraeus and his biographer, Paula Broadwell, both of whom are married, began several months after his retirement from the Army in August 2011 and ended four months ago, retired U.S. Army Col. Steve Boylan, a former Petraeus spokesman, told ABC News.

Broadwell, 40, had extraordinary access to the 60-year-old general during six trips she took to Afghanistan as his official biographer, a plum assignment for a novice writer.

"For him to allow the very first biography to be written about him, to be written by someone who had never written a book before, seemed very odd to me," former Petraeus aide Peter Mansoor told ABC News.

The timeline of the relationship, according to Patraeus, would mean that he was carrying on the affair for the majority of his tenure at the CIA, where he began as director Sept. 6, 2011. If he carried on the affair while serving in the Army, however, Patraeus could face charges, according to Article 134 of the Uniform Code of Military Justice, which reprimands conduct "of a nature to bring discredit upon the armed forces."

Whether the military would pursue such action, whatever evidence it accumulates, is unclear.

As the details of the investigation launched by the FBI unraveled this weekend, it became clear that the woman at the heart of the inquiry that led to Petraeus' downfall had been identified as Jill Kelley, a Florida woman who volunteers to help the military. She is a family friend of Petraeus, who Broadwell apparently felt threatened by.

Kelley and her husband are longtime supporters of the military, and six months ago she was named "Honorary Ambassador to Central Command" for her volunteer work with the military. Officials say Kelley is not romantically linked to Petraeus, but befriended the general and his wife when he was stationed in Florida. The Kelleys spent Christmases in group settings with the Petraeuses and visited them in Washington D.C., where Kelley's sister and her son live.

"We and our family have been friends with Gen. Petraeus and his family for over five years." Kelley said in a statement Sunday. "We respect his and his family's privacy and want the same for us and our three children."

Earlier this year, around the time that Petraeus and Broadwell were breaking off their affair, Kelly began receiving anonymous emails, which she found so threatening she went to authorities. The FBI traced the messages to Broadwell's computer, where they found other salacious and explicit emails between Broadwell and Petraeus that made it clear to officials that the two were carrying on an affair.

Investigators uncovered no compromising of classified information or criminal activity, sources familiar with the probe said, adding that all that was found was a lot of "human drama."

Broadwell, a married mother of two, had access to Petraeus while she was with him in Afghanistan as his official biographer. People close to the general had previously suspected Broadwell's feelings for him had crossed a professional line.

They found Broadwell, who spent a year embedded with Petraeus in Afghanistan, to be embarrassing and far too "gushy" about him. They said to one another they thought Broadwell "was in love with him," sources told ABC News.

Petraeus is said to have been the one to have broken off the extramarital affair.

His storied career, first as the public face of two wars in Iraq and Afghanistan, and later as director of the CIA, came crashing down Friday when he announced his resignation from the intelligence agency, citing the indiscretion.

"After being married for over 37 years, I showed extremely poor judgment by engaging in an extramarital affair. Such behavior is unacceptable, both as a husband and as the leader of an organization such as ours," Petraeus said in a statement Friday.

Director of National Intelligence James Clapper was made aware of the Petraeus situation Tuesday evening around 5 p.m. by the FBI, according to a senior intelligence source.

After having several conversations with Petraeus that evening and the next day, Clapper advised Petraeus that the best thing to do would be for him to resign, the source said.

Clapper notified the White House the next afternoon that Petraeus was considering resigning, according to the source. Petraeus then went to the White House Thursday and told the president he thought he should resign, and Obama accepted his resignation the next day, the source said.

Despite the lengthy investigation into Broadwell by the FBI, the White House says it was not made aware of it until Wednesday, the day after the election, a revelation that surprised many.

"It just doesn't add up. That the FBI would be carrying on this type of investigation without, again, bringing it to the president or the highest levels of the White House," Rep. Peter King, R-N.Y., said.

Petraeus and his wife, Holly, who have been married for 38 years, are said to be staying in their Arlington Home and are doing "OK."

"Knowing the family, I suspect it will be hard work, but given the effort, they will get through it," Boylan, the former Petraeus spokesman, said.

Numerous questions still remain about the investigation, and some on Capitol Hill are also frustrated because Petraeus was schedule to testify to the House and Senate intelligence committees about the attack on the U.S. consulate in Benghazi, Libya, in September.

The timing of Petraeus' resignation "was what it was," an official told ABC News, adding that the time had come to tie up any loose ends in the investigation and confront the general.

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Hathaway says ‘Les Mis’ made her feel deprived

NEW YORK (AP) — Anne Hathaway credits her new husband Adam Schulman for helping her get through the grueling filming of the screen adaptation of “Les Miserables.”

In “Les Mis,” the 30-year-old actress plays Fantine, a struggling, sickly mother forced into prostitution in 1800s Paris.

Hathaway lost 25 pounds and cut her hair for the role. She tells the December issue of Vogue, the part left her in a “state of deprivation, physical and emotional.” She felt easily overwhelmed and says Shulman was understanding and supportive.

The couple wed in September in Big Sur, Calif. Hathaway wore a custom gown by Valentino whom she collaborated with on the design. Working with the designer is a memory she says she will “treasure forever.”

The December issue of Vogue hits stores Nov. 20.

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Online:

http://www.vogue.com/

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Novartis drug helps patients with rare inflammatory diseases

ZURICH (Reuters) – Novartis‘ Ilaris helps reduce patients’ symptoms and the frequency of attacks in two rare inflammatory diseases, mid-stage studies showed, as the Swiss drugmaker looks to expand the use of the medicine.

Results of two separate studies on Sunday in patients with Familial Mediterranean Fever (FMF) and TRAPS – rare genetic diseases which can cause fever, rash and joint pain – both met their primary endpoints, Novartis said in a statement.

Both studies are being presented at the American College of Rheumatology (ACR) meeting in Washington D.C.

Ilaris or ACZ885, which blocks a protein called interleukin-1 beta that is thought to increase inflammation, is already sold for treating cryopyrin-associated periodic syndromes, a rare inflammatory disorder.

Novartis is also hoping to file the drug this year for regulatory approval in systemic juvenile idiopathic arthritis (SJIA), a debilitating disease that can affect a child’s growth.

Results of the phase II study showed the drug helped 100 percent of FMF patients reduce the frequency of attacks by at least 50 percent during three months of treatment.

Eight of the nine patients in the trial did not have an attack during the three months and blood markers of inflammation also normalized.

There are currently no approved treatments for FMF or TRAPS, rare genetically-inherited anti-inflammatory diseases, which can affect both children and adults.

Novartis is hoping to show the drug can be beneficial in treating rare inflammatory diseases after receiving a setback last year when U.S. health regulators rejected Ilaris for use in gout over concerns about side effects.

New data from a mid-stage study on the use of Ilaris in TRAPS showed that patients who came off therapy after being treated with the drug did not have a relapse for three months on average.

Earlier data from the study showed that 90 percent of patients experienced a significant improvement in symptoms after just one week of treatment with Ilaris. This rose to 95 percent after two weeks.

(Reporting by Caroline Copley; Editing by Elaine Hardcastle)

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Israel kills Gaza rocket crewman in second day of clashes

GAZA (Reuters) – An Israeli air strike killed a Palestinian militant in the Hamas-governed Gaza Strip on Sunday as a surge in cross-border violence entered its second day, local officials said.

Islamic Jihad, a smaller faction than Hamas which often operates independently, identified the dead man as one of its own, saying he was a member of a rocket crew hit by an Israeli missile in Jabalya, northern Gaza.

The Israeli military confirmed carrying out an air strike in the area. The death brought to six the number of Palestinians killed by Israel since four of its troops were hurt in a missile attack on their jeep along the Gaza boundary fence.

Islamic Jihad said it had fired 70 short-range rockets and mortar bombs across the border since Saturday, salvoes which drove Israeli residents to blast shelters. At least one Israeli, in the town of Sderot, was wounded, ambulance workers said.

Israel described the jeep ambush as part of a Palestinian strategy of trying to curb its countermeasures against possible cross-border infiltration. Israeli forces often mount hunts for tunnels and landmines on the inside of the Gaza boundary, creating a no-go zone for Palestinians.

“Of course we don’t accept their attempt to change the rules,” Defence Minister Ehud Barak told Israel’s Army Radio.

“The essence of the struggle is over the fence. We intend to enable the IDF (Israel Defence Forces) to work not just on our side but on the other side as well.”

Palestinians said four of Saturday’s dead were civilians hit by an Israeli tank shell while paying respects at a crowded mourning tent in Gaza’s Shijaia neighborhood. Israel denies targeting civilians.

The bloodshed puts internal pressure on Hamas, which, though hostile to the Jewish state, has sat out some of the recent rounds of violence as it tried to consolidate its Gaza rule and reach out to neighboring Egypt and other foreign powers.

Israel blames Hamas for any attacks emanating from Gaza, but has shown little appetite for a major sweep of the territory which might strain its own fraught ties to the new Islamist-rooted government in Cairo.

(Writing by Dan Williams; Reporting by Nidal al-Mughrabi; Editing by Todd Eastham)

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IBM surprised by Avantor lawsuit, calls claims exaggerated

(Reuters) – IBM, which is being sued by chemicals manufacturer Avantor Performance Materials for fraud and breach of contract in connection with a software project, said the accusations were blown out of proportion and that it was surprised by the move.

“We believe the allegations in the complaint are exaggerated and misguided and are surprised that Avantor chose to file suit,” the company said late on Friday in an emailed statement.

IBM said it had “met its contractual obligations and delivered a solution that Avantor continued to use in its operations.”

Avantor, which produces chemicals and raw materials for pharmaceutical products, laboratory chemicals and chemicals used in the electronics industry, filed a lawsuit on Thursday in the U.S. District Court for the District of New Jersey.

It said it was seeking tens of million in damages from IBM, which according to the lawsuit, had misrepresented the capabilities of a software program that runs on a platform by SAP, resulting “in a near standstill” of Avantor’s business.

(Reporting By Nicola Leske; Editing by Vicki Allen)

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Two senators restart talks on U.S. immigration reform plan

(Reuters) - Two key U.S. senators are restarting bipartisan talks on U.S. immigration reform that will include a path to citizenship for illegal immigrants already in the country, Senator Charles Schumer said on Sunday.

Appearing on NBC's "Meet the Press," the Democrat Schumer said he has spoken with Republican Senator Lindsey Graham and they have agreed to resume talks that broke off two years ago.

"And I think we have a darned good chance using this blueprint to get something done this year. The Republican Party has learned that being ... anti-immigrant doesn't work for them politically. And they know it," Schumer said.

President Barack Obama, re-elected last week with overwhelming support from Hispanic voters, in 2010 called the proposal unveiled by Graham and Schumer a "promising framework" on immigration reform. But the plan never got off the ground.

Obama's support among Hispanics was about 66 percent in the election, according to Reuters/Ipsos polling data.

There are an estimated 12 million illegal immigrants in the United States, most of them Hispanics.

The Graham and Schumer plan has four components: requiring high-tech, fraud-proof Social Security cards to ensure that illegal workers cannot get jobs; strengthening border security and enforcement of immigration laws; creating a process for admitting temporary workers; and implementing a path to legal status for immigrants already in the country.

Schumer said the plan embraces "a path to citizenship that's fair, which says you have to learn English, you have to go to the back of the line, you've got to have a job, and you can't commit crimes."

Many Republican leaders have taken a hard position against illegal immigrants. Obama's unsuccessful Republican challenger, Mitt Romney, said during the campaign that illegal immigrants should "self-deport" from the country.

Since the election, some influential conservative voices, including television commentator Sean Hannity, have announced support for immigration reform that includes a path to citizenship for illegal immigrants in the United States with no criminal record.

House of Representatives Speaker John Boehner, the top Republican in Congress, said on Friday that the U.S. immigration system is broken, and that Obama had to take the lead. Boehner has said he is confident Republicans could find common ground with the president.

The Obama administration announced in June it would relax U.S. deportation rules so that many young illegal immigrants who came to the United States as children can stay in the country and work.

The change would allow illegal immigrants who, among other criteria, are younger than 30 years old and have not been convicted of a felony to apply for work permits.

(Reporting by Will Dunham; Editing by Doina Chiacu)

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China says Hollywood putting pinch on domestic films

BEIJING (Reuters) – China‘s films are taking a hit from a trade deal that allows for more U.S. movie imports, the country’s broadcast regulator said on Sunday, with their share of the box office take sliding even as the industry’s total revenues outpace those of last year.

The movie pact, which exempted 14 films from China’s annual quota of 20 foreign films per year, was hammered out in February during a trip to the United States by Vice President Xi Jinping, the man expected to take the ruling Communist Party‘s top spot after a congress held this week in Beijing.

After signing the deal, the number of American films in China and their proportion of revenues have increased by a “large margin”, Vice Minister of the State Administration of Radio, Film and Television Tian Jin said.

“The past dominance of domestic films in the Chinese market has been shaken,” Tian told a press briefing on the sidelines of the congress held once every five years.

China’s 2012 box office revenues reached 13.27 billion yuan ($ 2.12 billion) at the end of October, Tian said, already outpacing revenues from all of 2011. But the share of revenues for domestically produced films was only 41.4 percent, constituting “a huge drop”.

Tian said the U.S. film industry is reaping massive profits while domestic producers are under greater pressure, mainly because Chinese movies cannot compete with the Hollywood spectacles.

“The competitiveness of Chinese-made films must be raised,” he said.

Chinese film industry experts have said that Hollywood’s looming shadow means Chinese producers need to focus on quality if they are going to elevate their appeal to a Chinese audience.

February’s deal stemmed from a victory in a 2009 U.S. World Trade Organization case that challenged Beijing’s restrictions on import and distribution of copyright-protected materials.

The U.S. movie industry has long complained about China’s tight restrictions on foreign films, which they say helps fuel demand for pirated DVDs that are widely available in China.

It also argued that it was being boxed out of a booming market, as the fast-growing Chinese middle class spends more money in theatres.

The Chinese film market is seen as one of the largest potential markets for Hollywood, but it has also been tightly controlled by the state-owned China Film Group.

Chinese films frequently compete for international awards, but winners overseas are often not those supported by China’s government, which tend to fan nationalist and patriotic sentiment.

(Reporting by Michael Martina; Editing by Nick Macfie)

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