Measuring the activity of a certain enzyme whose levels are low or absent in Fabry disease may be a useful newborn screening blood test for its diagnosis, a five-year study in Italy suggests. With the test, Fabry disease, which is more common among men than women, was found…
News
Fabry disease patients who have had a kidney transplant and are on immunosuppressive therapies may show a weaker response to a COVID-19 vaccine, including the booster, according to a study about two patients in France. While one patient had significant increases in antibody levels against SARS-CoV-2, the virus that…
Idorsia will continue its open-label extension of the Phase 3 MODIFY trial to better determine if lucerastat, the company’s investigational oral therapy for Fabry disease, may be of benefit to patients’ kidneys and the heart. The decision comes despite the therapy having failed to outperform a placebo…
Note: This story was updated Jan. 17, 2022, to note that Avrobio is deprioritizing its Fabry program. Avrobio is stopping its gene therapy program for Fabry disease in favor of clinical programs for other disorders, including cystinosis and Gaucher disease type 1. According to a company …
The Fabry International Network (FIN) is calling for global submissions for its annual FIN Award, which recognizes the efforts of patient organizations to educate and unify the Fabry disease community and to heighten awareness of the rare genetic lysosomal storage disorder. Projects must be initiated this year for…
A man living with Fabry disease for more than two decades was found to have a second co-occurring genetic condition called polycystic kidney disease, according to a recent report. “This case demonstrates the possibility of two [genetic kidney diseases] in the same individual and the risk of one diagnosis…
A single dose of 4D-310, an investigational gene therapy from 4D Molecular Therapeutics (4DMT), appears generally safe and restored the levels of alpha-galactosidase A (Gal A), the faulty enzyme in Fabry disease, in the first three men enrolled in a Phase 1/2 clinical trial. Notably, all three patients…
Jasper Therapeutics and Avrobio have entered into a non-exclusive research collaboration to evaluate the use of JSP191 in patients with Fabry disease who are being treated with one of Avrobio’s investigational gene therapies. Under this collaboration, JSP191 — an anti-CD117 monoclonal antibody — also will be…
Sangamo Therapeutics’ gene therapy candidate ST-920 safely and effectively increased and sustained the levels of the alpha-GalA protein, the enzyme that is lacking in people with Fabry disease, according to preliminary Phase 1/2 trial data. Four people have been treated at the first two therapy doses being…
FLT190, Freeline Therapeutics’ experimental gene therapy, led to sustained increases in alpha-galactosidase A (Gal A), the faulty enzyme in Fabry disease, in the first two patients given the one-time therapy in the Phase 1/2 MARVEL-1 clinical trial. Notably, the second patient, dosed early this year, has reached near-normal…
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