Publications by authors named "Daniel P Coller"
Article Synopsis
- Creatine transporter (CTD) and guanidinoacetate methyltransferase (GAMT) deficiencies cause serious brain issues like intellectual disabilities and seizures, with no effective treatment for CTD and a strict diet plus supplements needed for GAMT.
- A core outcome set (COS) has been developed in collaboration with caregivers and health professionals to determine key outcomes for assessing CTD and GAMT in clinical trials, including factors like cognitive functioning and emotional regulation.
- This COS aims to prioritize patient and caregiver perspectives to improve the drug development process, enhance trial comparability, reduce bias, and optimize resource use in research for these conditions.
