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2 articles matching "canavan disease"

ResearchCLINICALTRIALSApr 17

Trial Completed: Natural History Study of Patients With Canavan Disease (CANinform Study) (NCT04126005)

Researchers completed a study that collected information about how Canavan disease develops and progresses in children. The study looked at medical records from 67 patients and also gathered new information about their movement abilities and important health milestones. This type of study helps doctors understand the disease better and can guide future treatment development.

WHY IT MATTERSThis completed natural history study provides detailed data on how Canavan disease progresses in children, which is essential for designing future clinical trials and understanding what to expect from the disease.
ℹ Good to knowCanavan diseaseRead →
Clinical trialCLINICALTRIALSApr 17

Trial Now Recruiting: A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial) (NCT04998396)

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.
→ You can act on thisCanavan diseaseRead →

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