Patients’ anti-PEG antibodies may not block effects of Elfabrio: Study
About a third of Fabry disease patients were found to have low levels of antibodies against a substance called…
Articles by Lindsey Shapiro, PhD
Elfabrio soon available at low or no cost for Fabry patients in England
The National Institute for Health and Care Excellence (NICE) in England has recommended that Elfabrio (pegunigalsidase alfa) be covered…
High GI symptom burden in Fabry not linked to gut motility problems
Problems moving food through the gastrointestinal system didn’t largely account for gastrointestinal (GI) symptoms in Fabry disease patients, a…
Novel treatment shows promise in cell model of Fabry disease
Researchers have developed a novel treatment — an experimental substrate reduction therapy, or SRT — that may hold promise for…
Rare double mutations linked to case of cardiac Fabry in Japan
A rare pair of mutations were identified as the likely cause of Fabry disease for a young man with…
Optimized versions of Gal A enzyme may pave way to better treatments
Scientists have engineered versions of the alpha-galactosidase A (Gal A) enzyme that could eventually be used to produce a more…
Early Data for Gene Therapy FLT190 Support Ongoing MARVEL-1 Trial
FLT190, an experimental gene therapy from Freeline Therapeutics, safely led to stable increases in the activity of alpha-galactosidase…
Galafold Adherence High as Patients Report Life Quality Gains
Adults with Fabry disease exhibited high levels of adherence to at-home Galafold (migalastat) treatment over a two-year period, according…
Venglustat Lowers Gb3 With No Signs of Disease Progression in Trial
Daily treatment with the investigational oral therapy venglustat in men with Fabry disease led to significant reductions in…
Fabry GLA Gene Variant Found in Parkinson’s Patients
A variant in the GLA gene associated with Fabry disease was found in four women with Parkinson’s disease, but they…