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February 12, 2020February 14, 2020

Potential Fabry Treatment, PRX-102, Being Readied for Testing in Children

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Protalix BioTherapeutics and Chiesi Farmaceutici announced the start of a pediatric study plan (iPSP) to test PRX-102 (pegunigalsidase alfa) in children with Fabry disease, in agreement with the U.S. Food ... Read more

February 10, 2020February 10, 2020

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

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The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous ... Read more

February 5, 2020February 5, 2020

With Post-Brexit Move to Amsterdam Complete, EMA Renews Rare Disease Focus

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The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over ... Read more

January 29, 2020January 29, 2020

Fastex is ‘Better Tool’ to Measure Fabry Disease Progression Over Time in Individual Patients, Study Says

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A new evaluation tool, the FAbry STabilization indEX (Fastex), can help detect disease progression in individuals with Fabry disease, and could become useful in routine practice and clinical trial ... Read more

January 22, 2020January 22, 2020

No Race Component In Galafold Effectiveness, Subgroup Analysis Suggests

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Galafold (migalastat) is an effective treatment for certain Japanese people with Fabry disease, with no apparent differences in response to treatment due to race, a new analysis of data ... Read more

January 15, 2020January 15, 2020

Imaging Technique That Detects Changes in Eye Blood Vessels May Help in Early Diagnosis of Fabry, Study Says

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Detectable differences in blood vessel architecture in the eyes of people with Fabry disease could prove useful in diagnosing the disease early, a study has found. The study, “Optical ... Read more

January 8, 2020January 8, 2020

High-risk Screenings for Fabry Among Chronic Kidney Disease Patients Should Use Biomarkers Other Than Gb3, Study Says

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High-risk screenings for Fabry disease among individuals with chronic kidney disease (CKD) should be based on biomarkers other than globotriaosylceramide (Gb3) to lower the frequency of false positives, a ... Read more

December 18, 2019December 18, 2019

Cardiac Health Checks Should Be Given Women with GLA Mutations, Finish Study Suggests

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A common GLA mutation is highly associated with the development of Fabry disease in both men and women, and even women with no typical disease symptoms show signs of ... Read more

December 11, 2019December 10, 2019

Gene Therapy Takes Center Stage at 2019 NORD Summit

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With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD Rare Diseases & Orphan Products Breakthrough ... Read more

December 6, 2019December 6, 2019

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

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Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s ... Read more

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