Updates

May 24, 2019
Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries

RaDaR, the catchy new name for the U.S. government-run Rare Diseases Registry Program, aims to help patient advocacy groups with limited resources build their ... Read more
May 22, 2019May 22, 2019
Fabry Disease Unlikely to be Misdiagnosed for RA in Clinical Practice, Study Suggests

Fabry disease shares some manifestations with rheumatic diseases, which has raised concerns of wrongful diagnosis and consequent delayed treatment. Still, patients with this rare ... Read more
May 17, 2019May 15, 2019
Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns

With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose ... Read more
May 15, 2019May 15, 2019
FDA Approves Avrobio’s Gene Therapy Program for Fabry Disease

The U.S. Food and Drug Administration (FDA) has approved Avrobio’s clinical program for AVR-RD-01 as a gene therapy candidate for the treatment of Fabry ... Read more
May 1, 2019
PALS Uplifts the Spirits of Rare-Disease Patients by Offering Free Flights

Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult enough these days — even for healthy passengers. Imagine how ... Read more

News

Obstructive Sleep Apnea Not More Severe or Frequent in Patients with Mild Fabry Disease, Study Says
April 17, 2019April 17, 2019
Patients with mild Fabry disease do not have a higher prevalence or severity of obstructive sleep ...
News

Objective Cognitive Impairment Linked to Sex, Stroke, and IQ, Study Shows
April 10, 2019April 10, 2019
Male sex, history of stroke, and intelligence quotient (IQ) are associated with an increased ...
News

Female Patients and Their Doctors Must Be More Aware of Fabry Symptoms, Treatment, Study Says
April 3, 2019April 3, 2019
Although most women with Fabry disease have mild but typical manifestations, many are not prescribed ...
News

Galafold has Positive Effect on Fabry Patients with Amenable Mutations, Study Shows
March 27, 2019March 27, 2019
Amicus Therapeutics‘ Galafold (migalastat) provides clinical benefit to patients carrying amenable Fabry disease mutations, regardless ...
News

Fabry Disease More Common Than Previously Thought, Experts Say
March 20, 2019March 20, 2019
Two of the world’s top experts in Fabry disease, geneticists Dominique Germain of France and ...
News

Brexit Could Have Real Effects for UK Rare Disease Patients, Experts Warn
March 15, 2019March 14, 2019
Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will ...

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Galafold Improves Enzyme Activity and Heart Function in Patients With Amenable Fabry Disease Mutations, Study Says
March 13, 2019March 13, 2019
Galafold (migalastat) increases the activity of alpha-galactosidase A, stabilizes serum biomarkers, and improves heart ...
Moral Dilemmas Complicate Treatment of Rare Diseases, Says Israeli Bioethicist
March 13, 2019March 11, 2019
With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn ...
NIH Rare Disease Day Highlights Joint Networks Advancing Array of Research
March 6, 2019March 5, 2019
WODC 2019 Organizers Expect 1,200 to Attend Rare Disease Conference in April
February 27, 2019February 25, 2019
Vienna to Host RARE2019 Meeting on Rare Diseases
February 22, 2019February 20, 2019
Phase 3 Trial Shows Increased Stability of PRX-102 Over Other ERTs for Fabry Disease
February 13, 2019February 13, 2019

Gene Therapy AVR-RD-01 Shows Sustained Efficacy in Early Trial Data
February 12, 2019February 27, 2019
Treatment with the gene therapy candidate AVR-RD-01 showed sustained efficacy and was well-tolerated in ...
Patients, Family and Friends to Take Part in Variety of Rare Disease Day Events Worldwide
February 11, 2019
New Splicing Mutation in GLA Gene That Leads to Shorter Protein Found in Fabry Patient, Study Says
February 6, 2019February 6, 2019
ICD-10 Codes, ‘Really Important’ to Rare Disease Patients, Soon Up for Fresh Consideration
February 4, 2019February 6, 2019

FDA Revising ‘Draft Guidance’ on Developing Treatments for Rare Diseases
January 30, 2019January 30, 2019
The U.S. Food and Drug Administration(FDA) is updating its 2015 draft guidelines for drug ...
Enzyme Replacement Therapy Partially Restores Cellular Functions in Patients With Fabry and Gaucher Diseases, Study Finds
January 23, 2019January 23, 2019
Avrobio Earns FDA Orphan Drug Designation for Fabry Treatment AVR-RD-01
January 16, 2019January 16, 2019

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 ...
News

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 ...
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 ...

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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