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.
FDA approves first oral medication for treatment of adults with Fabry disease

FDA approves first oral medication for treatment of adults with Fabry disease

Canadian researchers use gene therapy to treat patient with Fabry disease

Canadian researchers use gene therapy to treat patient with Fabry disease

Review shows lipid nanoparticles as highly promising delivery systems in gene therapy

Review shows lipid nanoparticles as highly promising delivery systems in gene therapy

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

FDA clears Sangamo BioSciences' SB-318 IND application for treatment of MPS I

Neurocutaneous disorders affect skin, nervous system

Neurocutaneous disorders affect skin, nervous system

BfArM approves Greenovation’s moss-aGal phase I clinical trial for Fabry disease

BfArM approves Greenovation’s moss-aGal phase I clinical trial for Fabry disease

Synageva announces submission of Kanuma application to Cofepris for treatment of LAL Deficiency

Synageva announces submission of Kanuma application to Cofepris for treatment of LAL Deficiency

Fabry disease awareness: an interview with Dr. Hartmann Wellhoefer, Head of Medical Affairs, Rare Disease, Shire

Fabry disease awareness: an interview with Dr. Hartmann Wellhoefer, Head of Medical Affairs, Rare Disease, Shire

Study finds link between elevated urinary Gb3 and heart disease

Study finds link between elevated urinary Gb3 and heart disease

Genetic condition that attacks multiple organs can be detected using new MRI technique

Genetic condition that attacks multiple organs can be detected using new MRI technique

Genzyme marks 2013 International Rare Disease Day

Genzyme marks 2013 International Rare Disease Day

Sangamo BioSciences fourth quarter revenues increase to $8.9 million

Sangamo BioSciences fourth quarter revenues increase to $8.9 million

Researchers initiate first gene therapy clinical trial for Fabry disease

Researchers initiate first gene therapy clinical trial for Fabry disease

Researchers identify two new small 'chaperone' molecules that may treat Schindler/Kanzaki disease

Researchers identify two new small 'chaperone' molecules that may treat Schindler/Kanzaki disease

Shipment of Fabrazyme commences from Genzyme's FDA-and-EMA-approved Framingham plant

Shipment of Fabrazyme commences from Genzyme's FDA-and-EMA-approved Framingham plant

GSK, Angiochem to develop and commercialize LSD treatments

GSK, Angiochem to develop and commercialize LSD treatments

Researchers discover key interaction at heart of promising new treatment for Fabry disease

Researchers discover key interaction at heart of promising new treatment for Fabry disease

PDUFA goal date of NDA for Protalix's taliglucerase alfa extended

PDUFA goal date of NDA for Protalix's taliglucerase alfa extended

iBio receives U.S. patent entitled 'System for Expression of Genes in Plants'

iBio receives U.S. patent entitled 'System for Expression of Genes in Plants'