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December 4, 2018December 4, 2018

Fabry Stabilization Index (FASTEX) Deemed Valid, Specific for Patient Monitoring

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Fabry Stabilization index (FASTEX) is a valid, specific, and sensible instrument to accurately assess disease stability/progression in people with classic or non-classic Fabry disease, a large study showed. The ... Read more

November 27, 2018November 27, 2018

Replagal, an ERT, Appears Safe for Use During Pregnancy and Nursing, Case Study Reports

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Use of Replagal — an enzyme replacement therapy (ERT) — during pregnancy and breastfeeding by women with Fabry disease appears to be safe for them and their children, according to a ... Read more

November 20, 2018November 20, 2018

The Assistance Fund Now Offering Financial Help to Eligible Fabry Disease Patients

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To help reduce out-of-pocket medical expenses associated with managing and treating Fabry disease, an independent charitable patient assistance foundation has now added the disorder to its programs. Called The ... Read more

November 13, 2018November 13, 2018

ERT Dose Should be Tailored to Patient for Optimal Effectiveness, Study Shows

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Adjusting the dose of enzyme replacement therapy (ERT) for each patient may be essential to achieve complete neutralization of antibodies against the treatment, and reach better results to halt ... Read more

November 6, 2018November 6, 2018

Fabry Disease Symptoms’ Variability Goes Beyond Genetics, Report of 10 Cases Shows

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Fabry disease patients who share the same genetic mutation in the GLA gene may experience different symptoms, a case report study has found. The study, “Clinical Diversity in Patients with ... Read more

October 30, 2018October 29, 2018

#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach

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With the U.S. midterm elections now less than two weeks away, patient advocacy groups are solidly focused on a range of hot-button issues, from the Orphan Drug Tax Creditand ... Read more

October 30, 2018October 30, 2018

Absence of Lesions in Two Specific Brain Regions May Help Distinguish Fabry Disease from MS, Study Shows

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The absence of white matter lesions (WMLs) in two specific regions of the brain may help physicians distinguish Fabry disease from multiple sclerosis and make an accurate diagnosis, a ... Read more

October 25, 2018October 25, 2018

Young Fabry Disease Patients Show Brain Lesions in the Absence of Clinical History of Stroke

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Children and adolescents with Fabry disease who have no clinical history of stroke, show asymptomatic brain lesions, according to a follow-up neuroimaging study. Early detection of these lesions could make it easier for ... Read more

October 23, 2018October 23, 2018

Lantern Project Offers Diagnostic Opportunities for Rare Genetic Disease Patients

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Because there is relatively less awareness of rare genetic diseases, which often share symptoms common to better-known disorders, a program has been created to provide access to free diagnostic ... Read more

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

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