Fabry Disease More Common Than Previously Thought, Experts Say
Two of the world’s top experts in Fabry disease, geneticists Dominique Germain of France and Gheona Altarescu of Israel, say the number of people born with ... Read more
Updates
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Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn
Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as ... Read more -
Galafold Improves Enzyme Activity and Heart Function in Patients With Amenable Fabry Disease Mutations, Study Says
Galafold (migalastat) increases the activity of alpha-galactosidase A, stabilizes serum biomarkers, and improves heart function in patients with amenable Fabry disease mutations, according to ... Read more -
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist
With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases ... Read more -
NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research
Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the ... Read more
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WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April
The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government ... -
Vienna to Host RARE2019 Meeting on Rare Diseases
About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city ... -
Phase 3 Trial Shows Increased Stability of PRX-102 Over Other ERTs for Fabry Disease
PRX-102 (pegunigalsidase alfa) is safe for the treatment of patients with Fabry disease and ... -
Gene Therapy AVR-RD-01 Shows Sustained Efficacy in Early Trial Data
Treatment with the gene therapy candidate AVR-RD-01 showed sustained efficacy and was well-tolerated in ... -
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities ... -
New Splicing Mutation in GLA Gene That Leads to Shorter Protein Found in Fabry Patient, Study Says
Scientists discovered a new mutation in the GLA gene that leads to the production ...
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Protalix’s Pegunigalsidase Alfa Granted FDA Fast Track Status for Fabry Disease
News
The U.S. Food and Drug Administration (FDA) has granted fast track designation to pegunigalsidase alfa, or PRX-102, Protalix BioTherapeutics’ therapy candidate under development for Fabry disease. FDA’s fast track status is designed to facilitate the development and speed up FDA review ... Read more