Updates

August 21, 2019August 21, 2019
Amicus CEO John Crowley Outlines Progress in Pompe, Fabry, Batten Therapies

Pharmaceutical executives rarely make for a sympathetic Hollywood medical drama. But John Crowley did, and in the nearly 10 years since the release of ... Read more
August 20, 2019August 16, 2019
The Start of the Journey

Welcome to Morgan Yontz’s “Chronically Caring,” a new Fabry Disease News column. The first step I first learned of Fabry disease when my aunt tested ... Read more
August 19, 2019August 19, 2019
Nebraska’s Neena Nizar Seeks Cure for Jansen’s, One of World’s Rarest Diseases

Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one ... Read more
August 14, 2019August 14, 2019
Dried Blood Drops May Help Screen At-risk Patients Early for Fabry Disease, Study Suggests

Analysis of dry blood drops may help diagnose Fabry disease and begin early treatment in patients who are experiencing cardiac, renal, or neurological symptoms ... Read more
August 7, 2019August 7, 2019
Galafold Approved to Treat Fabry Teens and Adults in Argentina, Amicus Announces

Adolescents and adults with Fabry disease who live in Argentina can now be treated with Amicus Therapeutics’ oral precision medicine Galafold (migalastat). The Agency ... Read more

News

Oklahoma Ranks Lowest on Programs Key to Rare Diseases on NORD’s 2019 State Report Card
August 2, 2019August 1, 2019
Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of ...
News

Rare Disease Groups Seek Public Support to Renew Newborn Screening Act in Senate
August 1, 2019
Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, ...
News

FabryScan Screening Tool Shows Promise in Diagnosing Disease
July 31, 2019July 31, 2019
The screening tool FabryScan — consisting of a physical assessment and a questionnaire — ...
News

Gene Therapy AVR-RD-01 Effectively Prevents Buildup of Toxic Gb3 in Fabry Patients, Interim Data Show
July 24, 2019July 24, 2019
Avrobio’s investigational gene therapy AVR-RD-01 shows potential in promoting durable beneficial effects to treat ...
News

European Initiative Targets Diagnosis, Treatment of Rare Diseases
July 18, 2019
A new international consortium based in Paris, and funded largely by the 28-member European ...
News

Sperm Abnormalities in Men With Fabry Disease Don’t Compromise Fertility, Study Says
July 17, 2019July 17, 2019
Sperm abnormalities in men with Fabry disease do not compromise their fertility or overall ...

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Studying Rats May Make It Easier to Identify, Test Therapies for Fabry Eye Problems
July 10, 2019July 10, 2019
Rats that mimic Fabry disease develop eye symptoms like those observed in human patients. These ...
World’s First Alport Stamp Is Macedonian Mom’s Latest Win for Rare Disease Patients
July 4, 2019July 4, 2019
It wasn’t until Gordana Loleska’s son David was 14 years old that doctors in ...
uniQure’s Investigational Gene Therapy Shows Promise as Fabry Treatment
July 3, 2019July 3, 2019
Russian Nephrologists Largely Unaware About Fabry Disease; Patients with Kidney Failure Need Better Screening
June 26, 2019June 26, 2019
NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala
June 26, 2019June 25, 2019
Eurordis Unveils Integrated-care Initiative for Rare Disease Patients
June 21, 2019June 20, 2019

Protalix, Chiesi Preparing to Request Accelerated FDA Approval of PRX-102 for Fabry Disease
June 19, 2019June 19, 2019
Protalix BioTherapeutics and Chiesi Farmaceutici are planning to submit a biologics license application with ...
‘Rare Barometer’ Program Helps Eurordis Shape EU Rare Disease Policy
June 19, 2019June 17, 2019
Novel Gene Therapy Better at Targeting the Heart Shows Promise for Fabry Disease, Preclinical Data Suggest
June 12, 2019June 12, 2019
Patients Being Recruited in Europe to Test Gene Therapy Candidate FLT190 After Promising Preclinical Results
June 5, 2019June 5, 2019

Fabry Disease Gene Therapy ST-920 Well-tolerated, Shows Substantial Efficacy in Mice, Study Suggests
May 29, 2019May 29, 2019
One-time administration in mice of a gene therapy candidate known as ST-920 led to ...
Agency Unveils RaDaR to Help Patient Groups Develop Rare Disease Registries
May 24, 2019
Fabry Disease Unlikely to be Misdiagnosed for RA in Clinical Practice, Study Suggests
May 22, 2019May 22, 2019

News

Rare Diseases Constitute a ‘Public Health Issue,’ NCATS Director Warns
May 17, 2019May 15, 2019
With 250 rare diseases newly identified every year, scientists can barely keep up — ...
News

FDA Approves Avrobio’s Gene Therapy Program for Fabry Disease
May 15, 2019May 15, 2019
The U.S. Food and Drug Administration (FDA) has approved Avrobio’s clinical program for AVR-RD-01 ...
News

PALS Uplifts the Spirits of Rare-Disease Patients by Offering Free Flights
May 1, 2019
Cumbersome security procedures, rising airfares, and shrinking legroom have made commercial air travel difficult ...
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 ...

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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