Relay Therapeutics said it expects to start clinical development of its treatment candidate for Fabry disease in the second half of 2025. The program in Fabry disease, together with other programs under development for another genetic disease and solid tumors, were disclosed in a June 6 event, “New…
Fabry disease, in rare instances, may be among the causes of aseptic meningitis, a condition in which there is inflammation of the membranes covering the brain and spinal cord that’s not caused by an infection, a study found. The mechanisms underlying inflammation of these membranes, called the meninges, in…
A 69-year-old man diagnosed with Fabry disease who developed heart failure, high pulmonary arterial pressure, and heart microcirculation dysfunction saw his symptoms ease after treatment with vericiguat, according to a case reported in China. Vericiguat, which is sold as Verquvo, is an oral medicine that’s approved in the U.S.
A mutation in the GLA gene associated with Fabry disease was found by Italian researchers in a woman with end-stage kidney disease, according to a case report study. The 52-year-old woman was the sole family member with the disease-causing variant, meaning it was a de novo variant that wasn’t inherited…
Most children with Fabry disease, particularly those with the severe, classic form, have lower than normal skeletal muscle mass that affects the lower limbs more than the upper limbs, a study in China shows. Skeletal muscles are responsible for voluntary movements and play a vital role in everyday…
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…
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…
AceLink Therapeutics’ Phase 2 trial testing AL1211 as a treatment for Fabry disease has been cleared to launch by the U.S. Food and Drug Administration (FDA). The trial will evaluate AL1211’s safety and pharmacological properties in men diagnosed with classic Fabry disease who are willing to switch…
Periostin, a protein associated with kidney injury, may be a valuable biomarker of Fabry disease’s related kidney damage, according to new a study in Turkey. Researchers found that blood levels of periostin were correlated to proteinuria — the presence of proteins in the urine — which is one of…
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