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October 16, 2018October 16, 2018

Antioxidants May Help Prevent Damage to Heart, Kidneys in Fabry Patients, Research Suggests

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Inhibition of oxidative compounds using pharmacological or nutritional approaches, in addition to enzyme replacement therapy, may benefit patients with Fabry disease, research suggests. Treatment with antioxidant agents may help ... Read more

October 11, 2018October 11, 2018

Study Finds Correlation Between High Cholesterol and Cardiovascular Damage in Fabry Patients

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Cardiovascular damage is one of main causes of mortality in Fabry disease. A study now suggests that high HDL cholesterol levels are related to vascular lesions. The observational study, “Correlations ... Read more

October 9, 2018October 9, 2018

Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments

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Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced to seven years in federal prison for securities and wire fraud while heading San Diego-based ... Read more

October 4, 2018October 4, 2018

AVROBIO Reports Encouraging Preliminary Data for Phase 1/2 Trial Testing Gene Therapy in Fabry Patients

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AVROBIO, a biotechnology company developing single-dose gene therapies for rare diseases, announced positive preliminary data for its Phase 1/2 clinical trial testing AVR-RD-01 gene therapy in patients with Fabry ... Read more

October 2, 2018October 2, 2018

Screenings for Fabry Disease, Amyloidosis Provide Little Value in Polyneuropathy Patients, Study Suggests

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A study in a Nordic population found no apparent clinical benefit in screening patients with idiopathic small fiber neuropathy or mixed neuropathy for hereditary ATTR amyloidosis and Fabry disease, identifying ... Read more

September 27, 2018September 27, 2018

Stroke Linked to Fabry Disease After Check on Patient’s Family History

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A case study detailing a younger man admitted to a hospital for a stroke — later attributed to Fabry disease — highlights the importance of family history in diagnosing ... Read more

September 25, 2018September 24, 2018

#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington

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More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to converge on Washington, D.C., next month for the 2018 NORD Rare Diseases & Orphan Products ... Read more

September 20, 2018September 20, 2018

Intracranial Volume May Be Used as Measure of Brain Development in Fabry Disease, Study Finds

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The intracranial volume (ICV) in patients with Fabry disease is significantly lower than in healthy individuals, indicating an early onset of neurodevelopmental abnormality, according to researchers. Their study, “Reduced ... Read more

September 18, 2018September 18, 2018

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

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Protalix BioTherapeutics‘ investigational therapy PRX-102 (pegunigalsidase alfa) improved kidney function in Fabry disease patients, according to preliminary Phase 3 clinical results. The company announced that additional positive preliminary data of its BRIDGE clinical ... Read more

September 14, 2018September 14, 2018

White Matter Lesions Found in Nearly Half of Fabry Patients in Review Study, Especially Men

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Roughly half of the patients diagnosed with Fabry disease experience white matter lesions, which tend to develop earlier in males and are more prevalent as patients get older, a ... Read more