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Thursday, 21 January 2010 - Pompe disease hits screens in Extraordinary Measures |
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    Edition: U.S. Article Save Email Print Reprints Most Popular Most Shared China says "Avatar" most successful film at $80 million 20 Jan 2010 Obama to stick to agenda - White House adviser 20 Jan 2010 WRAPUP 5-Obama urges lawmakers "coalesce" on healthcare deal 20 Jan 2010 PRECIOUS-Gold steady a day after slump, China data eyed 20 Jan 2010 GLOBAL MARKETS-Dollar surges, Asia stocks slip after China data 20 Jan 2010 Salt reduction could save 92,000 U.S. lives a year 1:49am EST China Inc's global growing pains 19 Jan 2010 Apple to host event January 27, tablet expected 18 Jan 2010 Love in London is as rare as finding aliens 19 Jan 2010 Exclusive: Investors flee fund with ties to Galleon witness 20 Jan 2010 Pompe disease hits screens in "Extraordinary Measures" Toni Clarke BOSTON Wed Jan 20, 2010 9:14pm EST Related News Genzyme role in Pompe disease struggle hits theaters Wed, Jan 20 2010 A Minute With: Brendan Fraser's "Extraordinary Measures" Thu, Jan 14 2010 Genzyme revenue down on manufacturing woes Tue, Jan 12 2010 UPDATE 1-Genzyme names Ron Branning as SVP quality control Fri, Jan 8 2010 UPDATE 2-Genzyme, FDA agree a pathway for Lumizyme approval Thu, Dec 3 2009 BOSTON (Reuters) - "Extraordinary Measures", starring Harrison Ford and Brendan Fraser, opens on Friday and 101 employees from Genzyme Corp will gather in a Boston movie theater to see themselves portrayed in what some fear may be an unflattering light. Entertainment  |  Film The movie chronicles the story of John Crowley, currently the chief executive of Amicus Pharmaceuticals In, in his race to find a cure for Pompe disease, a rare muscle disorder that threatened to kill two of his three children. For the staff at Cambridge, Massachusetts-based Genzyme, which eventually developed a treatment, the movie has special meaning and opens as the biotechnology company struggles to emerge from its worst year ever. Last year, a manufacturing crisis caused it to close its Boston-area manufacturing plant after it strained capacity to breaking point. Ironically, it was the company's rush to ramp up production of the Pompe drug, called Myozyme, that led to the crisis. The movie is loosely based on the book "The Cure" by Wall Street Journal reporter Geeta Anand. It describes the emotional drama of the Crowleys' personal struggle to save their children, and the scientific drama of the drug's development. "This is our lives played out on screen, and we want to experience it together," said Lori Gorski, a Genzyme spokeswoman. Pompe disease affects some 5,000 to 10,000 people worldwide, and often kills babies before they reach the age of 2. People with the disease are deficient in an enzyme known as acid alpha-glucosidase, which is responsible for breaking down glycogen, a form of sugar stored in muscle cells. When glycogen builds up in these cells it can cause swelling of the heart and other organs and lead to disability and death. SCIENTIFIC BATTLE The race for a cure was fought out among scientists at four companies, including Genzyme. Between 1998 and 2002, Genzyme teamed up with a trio of companies and acquired rights to their experimental drugs. The company's first partnership was with Pharming Group NV, a company based in the Netherlands that was developing an enzyme using milk from hundreds of transgenic rabbits. Genzyme also partnered with a research group from Duke University that was using cells taken from Chinese hamster ovaries. In 2001 it acquired Novazyme, a company that John Crowley -- played by Brendan Fraser in the movie -- helped form based on the work of Dr. William Canfield, the scientist who is played by Harrison Ford. Genzyme also had its own product. Finally, Genzyme chief executive Henri Termeer demanded that his researchers stop bickering. "How many more babies have to die?" he shouted from the balcony of the company's boardroom. In late November 2001, Genzyme decided to run an experiment to figure out which program to focus on. A review of the data revealed Genzyme's product was the most efficacious and the easiest to manufacture. The other programs were discontinued and in 2006, Myozyme was approved in the United States and Europe. But the Pompe story was far from over. The U.S. Food and Drug Administration still has not approved a scaled-up version of the drug, which will be known in the United States as Lumizyme. That means there is still not enough of the commercial product to go around. Gorski thinks some employees might be hurt at the way Genzyme (called Zymagen in the film) is portrayed -- as a somewhat cold bureaucracy -- and said the real story is far more dramatic than any movie. "Putting 20 years of work into 90 minutes is a hard task that can never be put on screen," she said. (Reporting by Toni Clarke, editing by Maureen Bavdek) Entertainment Film More from Reuters Exclusive: Investors flee fund with ties to Galleon witness BOSTON (Reuters) - The tight-knit Boston hedge fund community has been rattled by a guilty plea in the biggest insider trading prosecution in two decades. Obama to target excessive financial risk-taking Asia stocks weak as China data signals tightening | Video Defense Secretary Gates arrives in Pakistan Exclusive: AIG owes retention payouts to many ex-employees How to make a play in the smart electrical grid: executives » More Top News Special Report: China Inc's growing pains China is overtaking Germany as the biggest exporting nation and has a car market bigger than America's. Now comes the hard part.  Full Article | Video  Factbox: China's outbound M&A Tough decisions loom for policymakers Davos High cost of "silent pandemic" Diseases like diabetes used to be dominant in the rich, but that's changing as the poor live longer. The shift could force drug companies to rethink their pricing.  Full Article  Glaxo offers free malaria research Malaria vaccine makes progress Pharmaceuticals © Copyright 2010 Thomson Reuters Editorial Editions: Africa Arabic Argentina Brazil Canada China France Germany India Italy Japan Latin America Mexico Russia Spain United Kingdom United States Reuters Contact Us Advertise With Us Help Journalism Handbook Archive Site Index Video Index   Analyst Research Mobile Newsletters RSS Podcasts Widgets Your View Labs Thomson Reuters Copyright Disclaimer Privacy Professional Products Professional Products Support Financial Products About Thomson Reuters Careers Online Products Acquisitions Monthly Buyouts.com Buyouts Europe: Buyouts Conferences: Venture Capital Journal ECVJ International Financing Review International Securitisation Report Project Finance International PEhub.com PE Week Thomson Reuters is the world's largest international multimedia news agency, providing investing news, world news, business news, technology news, headline news, small business news, news alerts, personal finance, stock market, and mutual funds information available on Reuters.com, video, mobile, and interactive television platforms. Thomson Reuters journalists are subject to an Editorial Handbook which requires fair presentation and disclosure of relevant interests. NYSE and AMEX quotes delayed by at least 20 minutes. Nasdaq delayed by at least 15 minutes. For a complete list of exchanges and delays, please click here.

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