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This article invites people to take a quiz about life expectancy in AAV (ANCA-associated vasculitis), a rare disease that affects blood vessels. After completing the quiz, you can see how your answers compare to other people who took it. The quiz is a way to learn more about what to expect with this condition.
WHY IT MATTERSUnderstanding life expectancy and prognosis in AAV can help patients and caregivers make informed decisions about treatment options and long-term planning.
Clinical trialCLINICALTRIALSApr 17
Researchers are testing a new gene therapy called BBP-812 to treat Canavan disease, a rare brain disorder that affects children. The therapy uses a modified virus to deliver a healthy copy of a gene that's missing or broken in people with this disease. This early-stage trial will check if the treatment is safe and whether it helps patients.
WHY IT MATTERSThis trial is now actively recruiting children with Canavan disease — if your child has been diagnosed, you may be eligible to participate in one of the first human tests of this gene therapy approach.
Clinical trialCLINICALTRIALSApr 14
Researchers tested a new gene therapy called ST-920 for Fabry disease in 36 patients. This treatment uses a modified virus to deliver instructions that help the body make an enzyme called alpha-galactosidase A, which people with Fabry disease don't produce enough of. The trial is now complete and tested whether different doses were safe and well-tolerated.
WHY IT MATTERSThis completed Phase 1/2 trial is the first human test of ST-920, meaning results could help determine if gene therapy can provide long-term relief for Fabry disease patients who currently require lifelong enzyme replacement infusions.