Top caregiver concerns in Rett syndrome and related disorders: data from the US natural history study

ConclusionThe top caregiver concerns for individuals with RTT and RTT-related disorders reflect the impact of the primary clinical symptoms of these disorders. This work is critical in the development of meaningful therapies, as optimal therapy should address these concerns. Further, outcome measures to be utilized in clinical trials should assess these clinical issues identified as most concerning by caregivers.
Source: Journal of Neurodevelopmental Disorders - Category: Neurology Source Type: research