Posts by: Marta Figueiredo

News

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, a large study showed. The ... Read more

November 27, 2018November 27, 2018

Replagal, an ERT, Appears Safe for Use During Pregnancy and Nursing, Case Study Reports

News

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

November 13, 2018November 13, 2018

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

News

Adjusting the dose of enzyme replacement therapy (ERT) for each patient may be essential to achieve complete neutralization of antibodies against the treatment, and reach better results to halt ... Read more

October 30, 2018October 30, 2018

Absence of Lesions in Two Specific Brain Regions May Help Distinguish Fabry Disease from MS, Study Shows

News

The absence of white matter lesions (WMLs) in two specific regions of the brain may help physicians distinguish Fabry disease from multiple sclerosis and make an accurate diagnosis, a ... Read more

September 18, 2018September 18, 2018

PRX-102 Improves Kidney Function in Fabry Patients, Preliminary Phase 3 Results Show

News

Protalix BioTherapeutics‘ investigational therapy PRX-102 (pegunigalsidase alfa) improved kidney function in Fabry disease patients, according to preliminary Phase 3 clinical results. The company announced that additional positive preliminary data of its BRIDGE clinical ... Read more

August 7, 2018August 7, 2018

Fabrazyme Biosimilar JR-051 Seen As Safe, Effective in Preclinical Animal Study

News

In a preclinical animal study, JCR Pharmaceuticals’ biosimilar candidate JR-051 was seen to be as effective as the approved therapy Fabrazyme (agalsidase beta) at reducing levels of globotriaosylceramide (Gb3), a fat molecule ... Read more

July 10, 2018July 10, 2018

European Experts Outline Organ-Specific Therapeutic Goals for Fabry Disease

News

A European panel of experts has defined a set of organ-specific therapeutic goals for Fabry disease, based on a systematic literature review and expert consensus. These therapeutic goals are ... Read more

June 26, 2018June 26, 2018

Late-onset Fabry Disease May Start Way Before Typical Signs, Study Shows

News

In late-onset Fabry disease, the accumulation of a fatty molecule in cells called Gb3 — and potential cellular damage — is present before typical signs of the disease, according ... Read more

June 14, 2018June 14, 2018

Researchers Discover Potential Predictive Profile of Heart Disease in Fabry Patients

News

Electrical, structural, and functional changes in the heart, along with abnormal accumulation of fat molecules, may predict the development of cardiac conditions in Fabry disease patients, according to researchers. Their ... Read more

May 8, 2018May 8, 2018

Liver Transplant Did Not Restore Fabry Patient’s GLA Levels, Case Study Finds

News

Transplanting the liver may not be an effective strategy to restore α-galactosidase A (GLA) levels — the deficient enzyme in Fabry disease patients — according to a case report. ... Read more