European Initiative Targets Diagnosis, Treatment of Rare Diseases
A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while ... Read more
Updates
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Sperm Abnormalities in Men With Fabry Disease Don’t Compromise Fertility, Study Says
Sperm abnormalities in men with Fabry disease do not compromise their fertility or overall reproductive function, a study finds. The findings of the study, ... Read more -
Studying Rats May Make It Easier to Identify, Test Therapies for Fabry Eye Problems
Rats that mimic Fabry disease develop eye symptoms like those observed in human patients. These animals can be used to test current and future therapies ... Read more -
World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in their native North Macedonia diagnosed his kidney, vision, and hearing ... Read more -
uniQure’s Investigational Gene Therapy Shows Promise as Fabry Treatment
AMT-190, uniQure‘s investigational gene therapy for the treatment of Fabry disease, shows promise as a one-time therapy option that may someday replace enzyme replacement ... Read more
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Russian Nephrologists Largely Unaware About Fabry Disease; Patients with Kidney Failure Need Better Screening
Few nephrologists in Russia are aware of Fabry disease, which is a problem that could ... -
NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala
A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy ... -
Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to ... -
Protalix, Chiesi Preparing to Request Accelerated FDA Approval of PRX-102 for Fabry Disease
Protalix BioTherapeutics and Chiesi Farmaceutici are planning to submit a biologics license application with ... -
Novel Gene Therapy Better at Targeting the Heart Shows Promise for Fabry Disease, Preclinical Data Suggest
A novel gene therapy developed by 4D Molecular Therapeutics can increase gene delivery to ...
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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