Updates

November 13, 2018November 13, 2018
ERT Dose Should be Tailored to Patient for Optimal Effectiveness, Study Shows

Adjusting the dose of enzyme replacement therapy (ERT) for each patient may be essential to achieve complete neutralization of antibodies against the treatment, and ... Read more
November 6, 2018November 6, 2018
Fabry Disease Symptoms’ Variability Goes Beyond Genetics, Report of 10 Cases Shows

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, ... Read more
October 30, 2018October 29, 2018
#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach

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 ... Read more
October 30, 2018October 30, 2018
Absence of Lesions in Two Specific Brain Regions May Help Distinguish Fabry Disease from MS, Study Shows

The absence of white matter lesions (WMLs) in two specific regions of the brain may help physicians distinguish Fabry disease from multiple sclerosis and ... Read more
October 25, 2018October 25, 2018
Young Fabry Disease Patients Show Brain Lesions in the Absence of Clinical History of Stroke

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 ... Read more

News

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 ...
News

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 ...
News

Study Finds Correlation Between High Cholesterol and Cardiovascular Damage in Fabry Patients
October 11, 2018October 11, 2018
Cardiovascular damage is one of main causes of mortality in Fabry disease. A study ...
News

Advocacy Groups, Doctors Question Rising Prices of Rare Disease Treatments
October 9, 2018October 9, 2018
Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced ...
News

AVROBIO Reports Encouraging Preliminary Data for Phase 1/2 Trial Testing Gene Therapy in Fabry Patients
October 4, 2018October 4, 2018
AVROBIO, a biotechnology company developing single-dose gene therapies for rare diseases, announced positive preliminary ...
News

Screenings for Fabry Disease, Amyloidosis Provide Little Value in Polyneuropathy Patients, Study Suggests
October 2, 2018October 2, 2018
A study in a Nordic population found no apparent clinical benefit in screening patients ...

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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
More than 700 medical experts, pharmaceutical executives, patient advocates, and others are expected to ...
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
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
Renal insufficiency in Fabry disease patients may lead to cellular events that result in ...
Heart Inflammation Prevalent in Fabry Disease Patients with Cardiomyopathy, Study Reports
September 4, 2018September 4, 2018
New Diagnostic Kit Can Screen Newborns for Fabry Disease Using Single Dried Blood Sample
August 28, 2018August 28, 2018
Researchers Create Fabry Heart Disease Model in a Culture Dish
August 23, 2018August 23, 2018

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 ...
Fabrazyme Biosimilar JR-051 Seen As Safe, Effective in Preclinical Animal Study
August 7, 2018August 7, 2018
Monitor Enzymes of Women With Fabry Disease to Protect Organs, Study Advises
August 2, 2018August 2, 2018

News

Chiesi Acquires Global Development, Sales Rights to PRX-102 as Treatment for Fabry Disease
July 31, 2018July 31, 2018
Chiesi, an Italian pharmaceutical company, has acquired the exclusive rights to develop and commercialize PRX-102 ...
News

Urine-derived Cells Are a Potential Diagnostic Tool for Fabry Disease, Researchers Say
July 23, 2018August 21, 2018
Urine-derived cells from patients with Fabry disease are a promising diagnostic tool and an adequate cellular model to ...
News

Experimental Lucerastat Suitable for All Types of Fabry Disease Patients, Study Shows
July 12, 2018July 12, 2018
Lucerastat, an investigative oral therapy currently in late-stage clinical development at Idorsia Pharmaceuticals, successfully reduced ...
News

European Experts Outline Organ-Specific Therapeutic Goals for Fabry Disease
July 10, 2018July 10, 2018
A European panel of experts has defined a set of organ-specific therapeutic goals for ...
News

Imaging Technique May Help Confirm Diagnosis of Fabry Disease, Case Study Reports
July 5, 2018July 5, 2018
A handheld imaging device may help physicians confirm a diagnosis of Fabry disease in ...
News

Changes in Gene Expression Linked to Pain, Cognitive Symptoms in Fabry Disease, Study Suggests
July 3, 2018July 3, 2018
Changes in gene expression in the prefrontal cortex, the area of the brain responsible ...

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