Author Archives: Joana Carvalho

Cerebellum Dysfunction May Contribute to Rett Motor Problems

Impairments in the cerebellum — a brain region responsible for controlling balance and movement coordination — contribute, in part, to motor impairments associated with Rett syndrome, a mouse study has found. These findings highlight the importance of understanding which brain areas contribute to the onset of specific symptoms associated…

Gilenya Fails to Ease Rett Symptoms in Phase 1/2 Trial

Despite being safe, one year of treatment with Gilenya (fingolimod) in young girls with Rett syndrome failed to ease such disease symptoms as lack of communication, motor impairments, and apraxia, or the inability to perform movements previously learned, a small Phase 1/2 trial showed. Moreover, the findings from the…

Small Molecule Aids Motor Skills, Breathing in Rett Mouse Model

Use of PTX-BD4-3, a small molecule that activates a protein receptor involved in a pathway that appears to be impaired in Rett syndrome, improved motor and respiratory function in a mouse model of the disease, a study found. These findings support further studies to better characterize PTX-BD4-3, possibly preparing…