The Latest Fabry Disease News

The latest Fabry Disease news from news providers and the NFDF.


Shire seeks approval to treat Fabry disease
Boston Globe, December 22, 2009 11:26 AM
 
British drugmaker Shire plc today said it has submitted a biologic license application to the Food and Drug Administration for Replagal, its treatment for the rare enzyme deficiency Fabry disease.  ... more



Diagnosis key in Roseburg man's recovery
NRToday.com - Roseburg,OR,USA

I put it together and thought maybe he does have (Fabry disease.)” Fabry disease occurs in about once in every 40000 people, Gray said.    ... more



Refocusing on Therapeutic Protein Production in Plants
Genetic Engineering News (press release)

Protalix has three other plant cell-produced enzymes in its preclinical pipeline: PRX-12, a galactosidase enzyme-replacement therapy for Fabry disease; ... ...more


Genzyme issues letters to healthcare providers reminding them of procedures for detection and filtration of potential foreign particles in Fabrazyme and other products.

Genzyme Press Release


Patients can help to ensure their own safety as we learn the news of potential particle contamination in vials of Fabrazyme (R).

As news emerges about the potential for small particles of materials from the manufacturing process to enter vials of our medication, we as patients can add a layer of safety by ensuring our infusions are completed using an in-line filter.

Jerry Walter, President, NFDF


Shire expands their team to respond to demand for information about access to Replagal (R).

Reportedly, in the recent past, physicians and patients contacting Shire's OnePath services that provides comprehensive support for patients, families and healthcare providers may have been unable to immediately reach a support provider. Busy phone lines due to the volume of requests for information caused some to reach voice mail instead of a support provider.

To ensure patients and physicians make prompt contact with a person who can provide support, Shire has expanded their team. Physicians, patients and family members seeking assistance can now get help without delay by contacting the OnePath services line at 1-866-888-0660. Fore more information about the OnePath services program, to obtain educational materials, to register for OnePath, or to contact Shire online, please visit http://www.onepath.com/.


Five Genzyme products for rare diseases found contaminated, but not a big danger
Los Angeles Times, November 15, 2009

Vials of five drugs produced by Genzyme Corp. of Framingham, Mass., have been found to be contaminated with tiny particles of steel, rubber or fiber, the Food and Drug Administration said Friday. ... more

More contamination troubles for Genzyme
Boston Globe, 14 November 2009

Cerezyme treats Gaucher disease, which causes fatty substances to accumulate in the liver, spleen, and other organs. Fabrazyme treats Fabry disease, ... ... more


The articles above are two of a series of articles emerging about potential particle contamination in Genzyme products. See links below to some of the other articles reported across the country:

FDA finds steel, rubber, other substances in Genzyme biotech drugs ...
Newser - USA

US FDA: Some Genzyme drugs contaminated
Interactive Investor - London, England, UK

More contamination found in Genzyme products
Boston Globe - Boston, MA

US FDA: Some Genzyme drugs contaminated
Reuters - USA



Shire Targets Fourth Quarter Filing of BLA for REPLAGAL(R) for Fabry Disease With U.S. FDA
CAMBRIDGE, Massachusetts, October 21 /PRNewswire

Early Access for Fabry Patients Available Under Treatment Protocol and Emergency IND

Shire announces plans to file a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) for REPLAGAL(R) (agalsidase alfa), its enzyme replacement therapy for Fabry disease, by the end of the year. ...more


6-year-old suffers through disease

Taylor Daily Press, Taylor Texas
Wednesday, October 21, 2009
By Jason Schaefer

For Brandon Yanez, every day is a struggle. There are good days and there are bad days, and on the bad days all he can do is cry.

Yanez is 6 years old, and suffers from a variety of muscular and joint problems. Doctors expect that by his 10th birthday, he will no longer be able to walk. ... more




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