A woman in her 20s with Fabry disease was safely treated with agalsidase alfa — a commonly used enzyme replacement therapy (ERT) — during her two pregnancies with no resulting complications, a case study reported. During the first pregnancy, “we did not observe any adverse event on…
Fabry Awareness Month, which is marked every April to draw attention to Fabry disease, is shining a light on the “heroes” who are living with the disease and those who support them. The month’s activities seek to heighten awareness among the general public, as well as among researchers,…
Centogene has extended a collaboration deal with Takeda in which the two companies will continue to provide diagnostic services to patients with lysosomal storage disorders — which may help people with Fabry disease to get a correct diagnosis. The partnership was initially established to improve patient access…
Researchers in England are working toward identifying and addressing barriers to reaching a Fabry disease diagnosis among underrepresented communities, particularly within ethnic minority and low socioeconomic status groups. The project is spearheaded by Richard Steeds, MD, a professor at the University of Birmingham and consultant cardiologist at University Hospitals…
Measures of heart function improved one to two years after treatment with 4D-310, an investigational gene therapy for Fabry disease, according to new trial data from 4D Molecular Therapeutics (4DMT), the therapy’s developer. “We are pleased to see 4D-310 continue to consistently demonstrate clinical activity across multiple important…
A smartphone app, called Fabry App, for remotely assessing Fabry disease was found by patients to be helpful for tracking day-to-day fluctuations in symptoms, while their doctors found it easier to monitor them and identify needs for intervention, a small study conducted in the U.K. shows. “A well-designed app can…
The investigational therapy AL01211 safely lowered levels of globotriaosylceramide (Gb3) — the molecule that toxically accumulates in Fabry disease — among healthy adults taking part in a Phase 1 trial, according to published results. The oral treatment, being developed by AceLink Therapeutics, also is being tested in…
A woman with Fabry disease was found to have two co-occurring disorders — a heart condition called dilated cardiomyopathy (DCM) and an autoimmune kidney disorder called immunoglobulin A nephropathy (IgAN) — a rare combination. “To the best of our knowledge, this is the first report on such a case,”…
The U.S. Food and Drug Administration (FDA) has advised Sangamo Therapeutics that positive results from a single well-controlled clinical trial, in addition to existing evidence, may be sufficient to form the basis for approving its Fabry disease gene therapy candidate. For Sangamo, that means that one…
The investigational gene therapy ST-920 (isaralgagene civaparvovec) has been well tolerated so far among adults with Fabry disease in the Phase 1/2 STAAR clinical trial, according to new interim data from its developer Sangamo Therapeutics. One-year data from more than a dozen…
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