Andrea Lobo, PhD, science writer —

A 10-year-old boy diagnosed with Fabry disease was also found to have a rare blood disorder called congenital dyserythropoietic anemia (CDA) in a case researchers said was highly unusual. In CDA, anemia, that is, a shortage of red blood cells that transport oxygen in the bloodstream, results in symptoms…

Chiesi Global Rare Diseases is launching a research grant initiative to support innovative studies into Fabry disease, alpha-mannosidosis, and cystinosis — all three of which are lysosomal storage disorders (LSDs). “Whilst the disorders can be rare individually, their prevalence is significant at a global level with an estimated…

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…