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July 10, 2019July 10, 2019

Studying Rats May Make It Easier to Identify, Test Therapies for Fabry Eye Problems

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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 for eye problems resulting from ... Read more

July 4, 2019July 4, 2019

World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients

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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 problems as Alport syndrome. Although ... Read more

July 3, 2019July 3, 2019

uniQure’s Investigational Gene Therapy Shows Promise as Fabry Treatment

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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 therapy (ERT) in this patient ... Read more

June 26, 2019June 26, 2019

Russian Nephrologists Largely Unaware About Fabry Disease; Patients with Kidney Failure Need Better Screening

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Few nephrologists in Russia are aware of Fabry disease, which is a problem that could be addressed by screening and diagnosing at-risk patients with end-stage renal disease who are undergoing hemodialysis. ... Read more

June 26, 2019June 25, 2019

NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala

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A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials ... Read more

June 21, 2019June 20, 2019

Eurordis Unveils Integrated-care Initiative for Rare Disease Patients

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Europe’s umbrella organization for 800 rare disease associations has developed a sweeping initiative to help the continent’s 30 million rare disease patients and their caregivers learn about their conditions, ... Read more

June 19, 2019June 19, 2019

Protalix, Chiesi Preparing to Request Accelerated FDA Approval of PRX-102 for Fabry Disease

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Protalix BioTherapeutics and Chiesi Farmaceutici are planning to submit a biologics license application with the U.S. Food and Drug Administration (FDA) for PRX-102 (pegunigalsidase alfa) as a treatment for ... Read more

June 19, 2019June 17, 2019

‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy

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People with rare diseases know that the right government policies can make a big difference in the quality of their own lives, and those of their caregivers. But most ... Read more

June 12, 2019June 12, 2019

Novel Gene Therapy Better at Targeting the Heart Shows Promise for Fabry Disease, Preclinical Data Suggest

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A novel gene therapy developed by 4D Molecular Therapeutics can increase gene delivery to the heart without triggering an immune response, according to preclinical data. The gene therapy candidate ... Read more

June 5, 2019June 5, 2019

Patients Being Recruited in Europe to Test Gene Therapy Candidate FLT190 After Promising Preclinical Results

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A single injection with candidate gene therapy FLT190 showed positive results in a mouse model of Fabry disease by significantly increasing blood levels of GLA — the enzyme affected ... Read more

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