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November 6, 2019November 6, 2019

Kidney Biopsy May Help Diagnose Female Fabry Patients, Case Report Suggests

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Kidney biopsies can be instrumental in diagnosing Fabry disease, particularly in females for whom symptoms can vary widely, a new case report contends. The report, “A case of female ... Read more

October 30, 2019October 30, 2019

Rapidly Aging Cells May Contribute to Shorter Lifespan of Fabry Patients, Study Finds

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Certain cells in patients with Fabry disease seem to age more rapidly than in healthy people, which may explain why patients have a shorter lifespan compared to the general ... Read more

October 23, 2019October 23, 2019

PRX-102 Improves Kidney Function in Fabry Patients, Preliminary Phase 3 Data Show

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Treatment with PRX-102 (pegunigalsidase alfa) can safely and effectively improve kidney function in Fabry disease patients, according to preliminary data from a Phase 3 clinical trial. One year of ... Read more

October 16, 2019October 16, 2019

‘Significant’ Levels of Pain Reported by Fabry Patients in Large Survey

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Even with treatment, people with Fabry disease often experience pain to an extent that it affects their quality of life, a study based on a survey of more than ... Read more

October 9, 2019October 9, 2019

Enrollment Completed in Phase 3 BALANCE Trial of PRX-102 for the Treatment of Fabry Disease

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Enrollment has been completed for a Phase 3 clinical trial comparing PRX-102 (pegunigalsidase alfa) with Fabrazyme (agalsidase beta) for the treatment of Fabry disease. The BALANCE trial (NCT02795676) estimated enrolling ... Read more

October 2, 2019October 2, 2019

Fabry Disease Patients with Higher Residual GLA Activity Respond Better to Galafold Treatment, Swiss Study Says

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Nearly half of Fabry patients have amenable or responsive mutations to Galafold (migalastat) but show different responses to the therapy, according to a Swiss population study. The study found ... Read more

September 27, 2019September 26, 2019

NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC

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Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board ... Read more

September 25, 2019September 25, 2019

Heart Cells Grown in Lab Dish May Help in Understanding Fabry’s Cardiac Complications

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Heart cells derived from patients’ stem cells and grown in a lab dish can reveal important clues about the development of heart ailments associated with Fabry disease. The study, ... Read more

September 18, 2019September 18, 2019

First Patient Dosed in Phase 1/2 Trial of Fabry Disease Gene Therapy Candidate FLT190

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The first patient in a Phase 1/2 clinical trial of Fabry disease gene therapy candidate FLT190 has been dosed. Enrollment is ongoing at the Royal Free Hospital, in London, ... Read more

September 4, 2019September 4, 2019

New GLA Mutation Leading to Low Enzyme Activity and Kidney Disease Found in Chinese Patient

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A new mutation in the GLA gene was found in a Chinese patient with Fabry disease that leads to low alpha-galactosidase A (alpha-GAL A) activity and correlates with kidney ... Read more

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