14th Feb 2024 Long-term benefits of enzyme replacement therapy in children and teenagers with Gaucher disease type 3- a plain language summary
17th Jan 2024 Looking at whether genetic testing can help doctors diagnose the severity of MPS I- a plain language summary
31st Jul 2023 Plain Language Summary: recommendations for managing diarrhea caused by trofinetide use in individuals with Rett Syndrome
31st Jan 2023 Plain language summary of a study looking at the age at diagnosis and time to start of treatment in individuals with mucopolysaccharidosis type I (MPS I)