Updates

February 13, 2019February 13, 2019
Phase 3 Trial Shows Increased Stability of PRX-102 Over Other ERTs for Fabry Disease

PRX-102 (pegunigalsidase alfa) is safe for the treatment of patients with Fabry disease and has better stability over other available therapies, according to interim ... Read more
February 11, 2019
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide

Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions ... Read more
February 6, 2019February 6, 2019
New Splicing Mutation in GLA Gene That Leads to Shorter Protein Found in Fabry Patient, Study Says

Scientists discovered a new mutation in the GLA gene that leads to the production of a shorter protein in a Chinese patient with Fabry ... Read more
February 4, 2019February 6, 2019
ICD-10 Codes, ‘Really Important’ to Rare Disease Patients, Soon Up for Fresh Consideration

G71.01 is, literally, the code for Duchenne muscular dystrophy. Q93.51 stands for Angelman syndrome, and G40.419 covers generalized and treatment-resistant epilepsies, which groups like ... Read more
January 30, 2019January 30, 2019
FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases

The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug discovery in rare diseases, with new guidance on natural history— ... Read more

News

Enzyme Replacement Therapy Partially Restores Cellular Functions in Patients With Fabry and Gaucher Diseases, Study Finds
January 23, 2019January 23, 2019
Enzyme replacement therapy (ERT) partially restores the function of lysosomes and mitochondria in patients ...
News

Avrobio Earns FDA Orphan Drug Designation for Fabry Treatment AVR-RD-01
January 16, 2019January 16, 2019
The U.S. Food And Drug Administration (FDA) has granted orphan drug status to Avrobio’s investigational gene therapy ...
News

Protalix BioTherapeutics Completes Enrollment for PRX-102 Phase 3 Trial
January 8, 2019January 8, 2019
Protalix BioTherapeutics announced it has completed enrollment of its Phase 3 trial evaluating PRX-102 in Fabry disease patients previously ...
News

Rising Healthcare Costs Strain EU Budgets Even as New Therapies Flourish
December 20, 2018December 20, 2018
Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures ...
News

LysoGb3 Blood Levels Can Help Diagnose Fabry Disease, Large Study Shows
December 18, 2018December 18, 2018
Globotriaosylsphingosine (LysoGb3) blood levels can be a useful and accurate biomarker to diagnose and ...
News

Fabry Stabilization Index (FASTEX) Deemed Valid, Specific for Patient Monitoring
December 4, 2018December 4, 2018
Fabry Stabilization index (FASTEX) is a valid, specific, and sensible instrument to accurately assess ...

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Replagal, an ERT, Appears Safe for Use During Pregnancy and Nursing, Case Study Reports
November 27, 2018November 27, 2018
Use of Replagal — an enzyme replacement therapy (ERT) — during pregnancy and breastfeeding by women ...
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 ...
ERT Dose Should be Tailored to Patient for Optimal Effectiveness, Study Shows
November 13, 2018November 13, 2018
Fabry Disease Symptoms’ Variability Goes Beyond Genetics, Report of 10 Cases Shows
November 6, 2018November 6, 2018
#NORDSummit – Major Issues on Table for Rare Disease Patients in US as Midterm Elections Approach
October 30, 2018October 29, 2018
Absence of Lesions in Two Specific Brain Regions May Help Distinguish Fabry Disease from MS, Study Shows
October 30, 2018October 30, 2018

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, ...
Lantern Project Offers Diagnostic Opportunities for Rare Genetic Disease Patients
October 23, 2018October 23, 2018
Antioxidants May Help Prevent Damage to Heart, Kidneys in Fabry Patients, Research Suggests
October 16, 2018October 16, 2018
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
Half a year has gone by since disgraced pharma executive Martin Shkreli was sentenced ...
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

News

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 ...
News

#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 ...
News

Intracranial Volume May Be Used as Measure of Brain Development in Fabry Disease, Study Finds
September 20, 2018September 20, 2018
The intracranial volume (ICV) in patients with Fabry disease is significantly lower than in ...
News

PRX-102 Improves Kidney Function in Fabry Patients, Preliminary Phase 3 Results Show
September 18, 2018September 18, 2018
Protalix BioTherapeutics‘ investigational therapy PRX-102 (pegunigalsidase alfa) improved kidney function in Fabry disease patients, according to preliminary ...
News

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 ...
News

Fabry Patients Show Impaired Exercise Capacity Due to Heart Function Deficits, Study Reports
September 11, 2018September 11, 2018
Fabry disease patients struggle with physical exercise — measured by a cardiopulmonary exercise test — ...

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

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