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

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

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

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

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

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

Research offers new hope for rare metabolic disorder

Research offers new hope for rare metabolic disorder

Simple genetic test can help in detecting heart abnormalities

Simple genetic test can help in detecting heart abnormalities

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

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

Shire submits REPLAGAL BLA with the FDA

Shire submits REPLAGAL BLA with the FDA

Genzyme ships vials of newly produced Cerezyme

Genzyme ships vials of newly produced Cerezyme

Shire announces third quarter 2009 financial results

Shire announces third quarter 2009 financial results

Modified NAGA: New enzyme for enzyme replacement therapy

Modified NAGA: New enzyme for enzyme replacement therapy

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

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

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

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

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

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

Genzyme receives Japanese marketing approval for Elaprase

Genzyme receives Japanese marketing approval for Elaprase

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

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

Novel preclinical gene therapy approach for treating Niemann-Pick disease

Novel preclinical gene therapy approach for treating Niemann-Pick disease

Stroke in young Fabry patients study

Stroke in young Fabry patients study

Drug shortfall as more than 5,000 conditions need medication

Drug shortfall as more than 5,000 conditions need medication

5,000 rare diseases need drugs, but Europe only approves a handful each year

5,000 rare diseases need drugs, but Europe only approves a handful each year