Fabry Disease News and Research

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Fabry disease (also known as Fabry's disease, Anderson-Fabry disease, angiokeratoma corporis diffusum and alpha-galactosidase A deficiency) is a rare X-linked recessive (inherited) lysosomal storage disease, which can cause a wide range of systemic symptoms.

Genetic Disorder Tracker introduced by Guidepoint Global

Guidepoint Global, LLC, a leading primary research firm, today announced the launch of their Genetic Disorder Tracker, a monthly longitudinal data release that captures commercial trends, on a worldwide basis, associated with the treatment of eight rare genetic disorders: Gaucher's disease, Fabry's disease, PKU, MPS I (Hurler Syndrome), MPS II (Hunter Syndrome), MPS VI (Maroteaux-Lamy Syndrome), Niemann-Pick disease and Pompe disease.

25 Feb 2010

Shire receives FDA Fast Track designation for REPLAGAL

Shire plc, the global specialty biopharmaceutical company, announces it has received Fast Track designation from the FDA for REPLAGAL(R) (agalsidase alfa), its enzyme replacement therapy for Fabry disease.

24 Feb 2010

Shire reports total revenues of $3,008 million for 2009

Shire plc the global specialty biopharmaceutical company, announces results for the year to December 31, 2009.

22 Feb 2010

Amicus Therapeutics presents additional data from Amigal Phase 2 extension study for Fabry disease

Amicus Therapeutics announced today additional positive preliminary data from its ongoing Phase 2 extension study of its investigational drug Amigal™ (migalastat HCl) for Fabry disease at the Lysosomal Disease Network WORLD Symposium in Miami, Florida.

11 Feb 2010

Shire's clinical study results for treatment of Gaucher disease to be presented at LDN World Symposium

Shire plc, the global specialty biopharmaceutical company, today announced that data from one of three Phase III clinical trials for velaglucerase alfa, the company's enzyme replacement therapy (ERT) in development for the treatment of Type 1 Gaucher disease, will be presented at the Lysosomal Disease Network (LDN) World Symposium, February 10-12, 2010 in Miami, Florida.

4 Feb 2010

Research offers new hope for rare metabolic disorder

A research team led by biochemist Scott Garman at the University of Massachusetts Amherst has for the first time determined the mechanism of one of the cell's "recycling" enzymes, human alpha-galactosidase or alpha-GAL, as it breaks down substances in the lysosome, the cell's recycling center.

2 Feb 2010

Simple genetic test can help in detecting heart abnormalities

Hypertrophic Cardiomyopathy (HCM) is a genetic disease that causes the heart muscle to become abnormally large. It often goes undetected and, as a result, HCM has the dubious distinction of being the leading cause of sudden death in children, young adults and athletes.

2 Feb 2010

Amicus Therapeutics presents corporate outlook for 2010 at Annual J.P. Morgan Healthcare Conference

Amicus Therapeutics today outlined the Company's three key strategic priorities and presented a corporate outlook for 2010 at the 28th Annual J.P. Morgan Healthcare Conference.

12 Jan 2010

Shire submits REPLAGAL BLA with the FDA

Shire plc , the global specialty biopharmaceutical company, today announced that it has submitted a BLA with the FDA for REPLAGAL(R) (agalsidase alfa), its enzyme replacement therapy for Fabry disease. REPLAGAL first received marketing authorization in the European Union in 2001, and is approved for the treatment of Fabry disease in 45 countries.

22 Dec 2009

Genzyme ships vials of newly produced Cerezyme

Genzyme Corporation announced today that it has begun shipping vials of newly produced Cerezyme® (imiglucerase for injection) manufactured at its Allston Landing plant.

2 Dec 2009

Shire announces third quarter 2009 financial results

Shire plc, the global specialty biopharmaceutical company, announces results for the three months to September 30, 2009.

31 Oct 2009

Modified NAGA: New enzyme for enzyme replacement therapy

A new study uses a creative structure-based remodeling strategy to design a therapeutic protein that exhibits significant advantages over currently available treatments for a rare disease that often leads to cardiac and renal failure. The research, published by Cell Press on October 22nd in the American Journal of Human Genetics, describes a new and highly promising candidate for enzyme replacement therapy (ERT) for Fabry disease.

23 Oct 2009

Shire to file a Biologics License Application for REPLAGAL with the FDA

Shire plc, the global specialty biopharmaceutical company, 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.

21 Oct 2009

Enzyme therapy with agalsidase alfa may slow the progression of kidney disease

For men with Fabry disease, enzyme replacement therapy (ERT) with agalsidase alfa slows deterioration of kidney function, reports a study in the online edition of the Journal of the American Society of Nephrology (JASN).

9 Apr 2009

Amicus Therapeutics presents positive results from phase 2 extension study of Amigal for Fabry disease

Amicus Therapeutics has announced positive results from its ongoing Phase 2 extension study of its investigational drug, Amigal (migalastat HCL) for Fabry disease.

29 Mar 2009

Genzyme receives Japanese marketing approval for Elaprase

Genzyme Corp. has announced that it has received approval to market Elaprase (idursulfase) in Japan for the treatment of Hunter syndrome.

4 Oct 2007

Combination of blood pressure-lowering drugs stops loss of kidney function in rare genetic disease

A combination of two types of blood pressure-lowering drugs, an angiotensin-converting enzyme inhibitor (ACEI) plus an angiotensin-receptor blocker (ARB), added to enzyme replacement therapy (ERT) with agalsidase-beta (Fabrazyme, Genzyme Corporation, Cambridge, MA) is the first treatment shown to stop progressive loss of kidney function in patients with severe kidney involvement due to the rare genetic disorder Fabry disease, reports a study in the September Journal of the American Society of Nephrology.

26 Jul 2007