Messenger RNA (mRNA) treatment restored levels of Gal A enzyme activity in heart cells derived from two Fabry disease patients with severe heart involvement, a study showed. The mRNA therapeutic delivered the correct information contained in the GLA gene to produce a fully functional Gal A enzyme, which is…
A team of expert clinicians and patient advocates has created a new set of recommendations to guide the use of Galafold (migalastat) in people with Fabry disease, highlighting the importance of centering patients’ preferences in treatment decisions. “We hope that this publication will lead to the provision of…
The duration and rate of Fabrazyme (agalsidase beta) infusion can be safely changed in people with Fabry disease, independently of their body weight, if appropriate care is taken, according to a new study from Japan. Specifically, an infusion time shorter than 90 minutes or an infusion rate higher…
A man diagnosed in his 60s with late-onset Fabry disease had an “uneventful” recovery after receiving both a heart and a kidney transplant in surgeries seven years apart, according to a new case report. “Heart and kidney transplants can play a major role in patients with Fabry disease who develop…
The National Institute for Health and Care Excellence (NICE) in England has recommended that Elfabrio (pegunigalsidase alfa) be covered by the country’s national health service (NHS) and provided at low or no cost to adults with Fabry disease. About 1,150 people in England are estimated to have Fabry,…
People with Fabry disease who have burning limb pain or depression are more likely to report poor quality of life, a new study shows. Addressing these issues may help them, researchers said in “Screening for health-related quality of life and its determinants in Fabry disease: A…
Problems moving food through the gastrointestinal system didn’t largely account for gastrointestinal (GI) symptoms in Fabry disease patients, a recent study shows. The symptoms, which have an impact on patients’ quality of life, may instead arise from dysfunction in the nerve cells that control gut reflexes. The study, “…
Chiesi Global Rare Diseases and Aliada Therapeutics are teaming up to advance a blood-brain barrier (BBB) crossing platform technology to deliver therapies for lysosomal storage disorders, including Fabry disease. The BBB is a semipermeable membrane that limits what substances can pass from the bloodstream into the brain.
AceLink Therapeutics has opened its first clinical site in China for a Phase 2 clinical trial testing AL01211 as a treatment for Fabry disease. The trial is actively screening and enrolling patients across six sites in China, including Shanghai’s Ruijin Hospital. Five other sites are expected to open by…
A gene therapy treatment increased levels of the deficient alpha-glactosidase A (Gal A) enzyme in the organs of mice in a model of Fabry disease, according to a new study. Results showed the gene therapy approach reduced the toxic buildup of fatty molecules in most organs assessed, but not…
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