Updates

January 8, 2019January 8, 2019
Protalix BioTherapeutics Completes Enrollment for PRX-102 Phase 3 Trial

Protalix BioTherapeutics announced it has completed enrollment of its Phase 3 trial evaluating PRX-102 in Fabry disease patients previously treated with Shire’s Replagal (agalsidase alfa). The BRIDGE trial (NCT03018730) is an ... Read more
December 20, 2018December 20, 2018
Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish

Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of ... Read more
December 18, 2018December 18, 2018
LysoGb3 Blood Levels Can Help Diagnose Fabry Disease, Large Study Shows

Globotriaosylsphingosine (LysoGb3) blood levels can be a useful and accurate biomarker to diagnose and monitor Fabry disease, rather than only using GLA mutational status, ... Read more
December 4, 2018December 4, 2018
Fabry Stabilization Index (FASTEX) Deemed Valid, Specific for Patient Monitoring

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, ... Read more
November 27, 2018November 27, 2018
Replagal, an ERT, Appears Safe for Use During Pregnancy and Nursing, Case Study Reports

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

News

The Assistance Fund Now Offering Financial Help to Eligible Fabry Disease Patients
November 20, 2018November 20, 2018
To help reduce out-of-pocket medical expenses associated with managing and treating Fabry disease, an ...
News

ERT Dose Should be Tailored to Patient for Optimal Effectiveness, Study Shows
November 13, 2018November 13, 2018
Adjusting the dose of enzyme replacement therapy (ERT) for each patient may be essential ...
News

Fabry Disease Symptoms’ Variability Goes Beyond Genetics, Report of 10 Cases Shows
November 6, 2018November 6, 2018
Fabry disease patients who share the same genetic mutation in the GLA gene may ...
News

#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach
October 30, 2018October 29, 2018
With the U.S. midterm elections now less than two weeks away, patient advocacy groups ...
News

Absence of Lesions in Two Specific Brain Regions May Help Distinguish Fabry Disease from MS, Study Shows
October 30, 2018October 30, 2018
The absence of white matter lesions (WMLs) in two specific regions of the brain ...
News

Young Fabry Disease Patients Show Brain Lesions in the Absence of Clinical History of Stroke
October 25, 2018October 25, 2018
Children and adolescents with Fabry disease who have no clinical history of stroke, show asymptomatic brain lesions, ...

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Lantern Project Offers Diagnostic Opportunities for Rare Genetic Disease Patients
October 23, 2018October 23, 2018
Because there is relatively less awareness of rare genetic diseases, which often share symptoms ...
Antioxidants May Help Prevent Damage to Heart, Kidneys in Fabry Patients, Research Suggests
October 16, 2018October 16, 2018
Inhibition of oxidative compounds using pharmacological or nutritional approaches, in addition to enzyme replacement ...
Study Finds Correlation Between High Cholesterol and Cardiovascular Damage in Fabry Patients
October 11, 2018October 11, 2018
Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments
October 9, 2018October 9, 2018
AVROBIO Reports Encouraging Preliminary Data for Phase 1/2 Trial Testing Gene Therapy in Fabry Patients
October 4, 2018October 4, 2018
Screenings for Fabry Disease, Amyloidosis Provide Little Value in Polyneuropathy Patients, Study Suggests
October 2, 2018October 2, 2018

Stroke Linked to Fabry Disease After Check on Patient’s Family History
September 27, 2018September 27, 2018
A case study detailing a younger man admitted to a hospital for a stroke ...
#NORDSummit – More Than 700 Expected to Attend Oct. 15-16 Rare Disease Summit in Washington
September 25, 2018September 24, 2018
Intracranial Volume May Be Used as Measure of Brain Development in Fabry Disease, Study Finds
September 20, 2018September 20, 2018
PRX-102 Improves Kidney Function in Fabry Patients, Preliminary Phase 3 Results Show
September 18, 2018September 18, 2018

White Matter Lesions Found in Nearly Half of Fabry Patients in Review Study, Especially Men
September 14, 2018September 14, 2018
Roughly half of the patients diagnosed with Fabry disease experience white matter lesions, which ...
Fabry Patients Show Impaired Exercise Capacity Due to Heart Function Deficits, Study Reports
September 11, 2018September 11, 2018
Renal Insufficiency in Fabry Patients May Lead to Cardiovascular Disease, Study Shows
September 7, 2018September 7, 2018

News

Heart Inflammation Prevalent in Fabry Disease Patients with Cardiomyopathy, Study Reports
September 4, 2018September 4, 2018
Inflammation of the heart, known as myocarditis, is found in more than half of ...
News

New Diagnostic Kit Can Screen Newborns for Fabry Disease Using Single Dried Blood Sample
August 28, 2018August 28, 2018
A new diagnostic tool that can screen for up to six lysosomal storage disorders, ...
News

Researchers Create Fabry Heart Disease Model in a Culture Dish
August 23, 2018August 23, 2018
Japanese researchers have used stem cells from Fabry patients to create an innovative laboratory ...
News

FDA Gives Galafold Accelerated Approval for Treatment of Fabry Patients
August 14, 2018August 14, 2018
The U.S. Food and Drug Administration has granted accelerated approval to Galafold (migalastat) 123 mg capsules to ...
News

Fabrazyme Biosimilar JR-051 Seen As Safe, Effective in Preclinical Animal Study
August 7, 2018August 7, 2018
In a preclinical animal study, JCR Pharmaceuticals’ biosimilar candidate JR-051 was seen to be as ...
News

Monitor Enzymes of Women With Fabry Disease to Protect Organs, Study Advises
August 2, 2018August 2, 2018
Women with Fabry disease can suffer from significant disease that affects several organs, and ...

February 6, 2018February 6, 2018

Protalix’s Pegunigalsidase Alfa Granted FDA Fast Track Status for Fabry Disease

News

The U.S. Food and Drug Administration (FDA) has granted fast track designation to pegunigalsidase alfa, or PRX-102, Protalix BioTherapeutics’ therapy candidate under development for Fabry disease. FDA’s fast track status is designed to facilitate the development and speed up FDA review ... Read more