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Pompe Canada
Walk with Team Pompe for Muscular Dystrophy PDF Print E-mail
articles - Ontario news
Written by Tyler Reilly   
Wednesday, 09 June 2010 15:50

Walk with Team Pompe for Muscular Dystrophy

Date:
Sunday, June 13, 2010
Time:
11:00am - 2:00pm
Location:
Bay Front park in Hamilton
Hi everyone it is that time of year again for the Hamilton Walk for Muscular Dystrophy at Bay Front park Sunday June 13th. Hopefully some of you will join us again on the walk as it is allways a fun day. If you wish to sponsor our team please follow this web link http://my.e2rm.com/TeamPage.aspx?teamID=161863&langPref=en-CA

Thanks,
Ian

 

 
Brad's fight against Pompe Disease - a silent killer PDF Print E-mail
articles - getting myozyme
Written by John Crittenden   
Wednesday, 10 February 2010 19:03

Brad's fight against Pompe Disease - a silent killer

Second letter: February 10, 2010: TO THE HONOURABLE KEVIN FALCON, BC MINSTER OF HEALTH:

Dear Kevin,

This is my second letter to you and the reason is that I want to clearly identify the life and death situation my son faces so that I know that you know the seriousness of this.

• Brad has adult-onset Pompe disease which will take his life if not treated immediately.

• Myozyme therapy for Pompe has been approved by Health Canada and adult-onset Pompe therapy is funded in Alberta and Ontario. It is now saving the lives of adult-onset Pompe patients in both provinces but has not been made available to Brad, who lives in British Columbia, because BC has not funded it. We have been told that Brad is the only adult-onset Pompe patient in BC that would benefit from this therapy.

Brad's condition is deteriorating and time is of the essence. He has already lost one diaphragm. If he loses the other diaphragm he will face the rest of a short life bedridden, on a respirator, with a feeding tube, and die a disgusting and cruel death.

Brad's doctor has applied for funding for a two year trial for Brad which would provide the treatment he needs. That application is tied up in Pharmacare and I was told by a person at the Ministry of Health that it will be months before a decision is made. By then it will be too late to save Brad.

You have the power to do something about this. What would you do if Brad were your son?

Please don't let this happen. Approve treatment on a compassionate basis. There is not a person in this province that would fault you. Quite the contrary. It is the right thing to do.

Sincerely,
John Crittenden

Attachment: Brad's request for Myozyme report (PDF)
Medical report: Pompe LOTS Early Absract Laforet (PDF)
Medical report: Survey2-AusPatientsOnMyozyme-July2009-FINAL (2) (PDF)
Medical report: Pompe respiratory Munich Khan.pdf (PDF)

 
National CBC News PDF Print E-mail
articles - extraordinary measures
Written by Tyler Reilly   
Sunday, 24 January 2010 00:20

Here is the link:
http://www.cbc.ca/thenational/watch/

The first Acknowledgement is 12min. in

the main link is 14min. into prodcast

 
Evening News Expensive treatment PDF Print E-mail
articles - getting myozyme
Written by Tyler Reilly   
Thursday, 28 January 2010 09:16

Evening News

Expensive treatment

An Okanagan man is fighting for medical coverage for a disease so rare he says only one other person in B.C. has it.

 

http://www.chbcnews.ca/video/index.html

 
Genzyme chief expects to increase Mass. workforce by at least 500 PDF Print E-mail
articles - extraordinary measures
Written by Robert Weisman, Globe Staff   
Friday, 22 January 2010 20:13

Genzyme chief expects to increase Mass. workforce by at least 500

The Boston Globe

 

By Robert Weisman, Globe Staff | January 22, 2010

 

Genzyme Corp.<http://finance.boston.com/boston?Page=QUOTE&Ticker=GENZ>, which is working to end rationing of two key drugs and return to full-scale production by midyear at its troubled Allston Landing plant, expects to hire more than 500 workers in Massachusetts this year, mostly at its headquarters in Cambridge and at manufacturing sites in Framingham and Allston.

The biotechnology company’s plans were disclosed by chief executive Henri Termeer during a visit to the Globe yesterday. Termeer said the company added 330 employees in the state last year and 1,000 globally, boosting its workforce to about 12,000, including 4,500 in Massachusetts. The number of employees could double over the next five years, as Genzyme<http://finance.boston.com/boston?Page=QUOTE&Ticker=GENZ> brings new drugs into the marketplace, he said.

Termeer, in a wide-ranging discussion, also said he is not deterred by a pair of activist investors who have been buying Genzyme stock, expressed his disappointment in the blow dealt to the health care overhaul by this week’s Senate election in Massachusetts, and said he is determined to put Genzyme’s production problems behind him.

“We are at the beginning of the turnaround, and it looks pretty encouraging,’’ Termeer said, citing progress made in Allston.

While the company had three drugs approved in 2009, the year was dominated by its struggle to correct production defects cited by federal regulators and clean up after a virus was found in a bioreactor at the Allston plant. The virus forced Genzyme to suspend manufacturing temporarily and ration two costly enzyme replacement drugs that treat rare genetic disorders: Cerezyme for Gaucher disease, which causes swelling of the liver, spleen, and other organs; and Fabrazyme for Fabry disease, which causes the build-up of fat in eyes and kidneys.

Genzyme resumed full delivery of Cerezyme in December and expects to supply 80 to 100 percent of required doses for Fabrazyme patients by the end of the second quarter, Termeer said.

Much of the hiring in Massachusetts last year and this year will be to staff a new plant in Framingham built to increase the company’s capacity to make Cerezyme and Fabrazyme so it will not be caught short in the event of another emergency. Framingham received a $12.9 million state grant to make infrastructure improvements, adding water and sewer lines, to accommodate the Genzyme plant and other operations under Governor Deval Patrick’s $1 billion initiative to promote life sciences.

At a separate Framingham plant, Genzyme is producing the drug Myozyme to treat children younger than 18 who suffer from Pompe disease, a rare genetic disorder that interferes with muscle development and causes severe respiratory problems.

Genzyme said yesterday that the Food and Drug Administration has set June 17 as its decision date on the company’s application, filed late last year, to make a variant of that drug, called Lumizyme, on a larger scale for adult Pompe disease patients. That drug is the most important of several in the company’s pipeline that could expand its revenue and employment base in the first half of the new decade, though production of Lumizyme will take place in Belgium.

“Lumizyme looks poised for a mid-2010 approval,’’ Christopher J. Raymond, senior biotechnology analyst for Chicago financial firm Robert W. Baird & Co., wrote in a note to investors yesterday. But he cautioned that Genzyme faces intense competition from rivals to Cerezyme, its top-selling product, despite a 10 percent increase in its share price since the start of the year. Shares closed down 42 cents, or 0.7 percent, yesterday to $54.43 on the Nasdaq exchange.

Termeer said he was confident two shareholder activists, Ralph Whitworth of California and Carl C. Icahn of New York, have been accumulating Genzyme shares because they see it as a good investment, not because they have designs on the company.

He acknowledged he has not met with Icahn, a billionaire investor who has taken a 1 percent stake in Genzyme. The company has promised to offer Whitworth, cofounder of San Diego asset management firm Relational Investors LLC, which has amassed a 4 percent stake, a seat on Genzyme’s board next fall as part of a mutual cooperation pact.

“Ralph is interested in a return for his investment capital,’’ Termeer said. “Carl is in it for the investment. Our stock has been trading down. He made an investment, like a lot of other people. He will get a good return on it. . . . His most important thing is not to create real disruption; it’s to create a real return. And if he studies it, he will find the best way to create real return is to let us recover.’’

Termeer also said that he supports an overhaul of the federal health care system and was disheartened that movement on a comprehensive national health care bill appears to have been derailed by Republican Scott Brown’s election to the Senate.

But he suggested a provision important to the biotech industry, granting 12-year data exclusivity for drugs before generic versions are allowed on the market, still could be approved in a separate bill.

Robert Weisman can be reached at weisman@globe.com



Last Updated on Saturday, 23 January 2010 20:02
 
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