Matthew Gavidia

Patients with the GBA mutation of Parkinson disease (PD) were shown to exhibit more severe cognitive decline than patients with idiopathic PD and those with both the GBA and LRRK2 G2019S mutations of PD.

In adolescent patients with sickle cell disease, hydroxycarbamide therapy was associated with improvements in neurocognition variables such as working memory, verbal memory, and nonverbal IQ, according to study findings.

The Institute for Clinical and Economic Review (ICER) published a draft evidence report on crizanlizumab (Adakveo), voxelotor (Oxbryta), and L-glutamine (Endari) for sickle cell disease, as well as a draft scoping document on valoctocogene roxaparvovec, an investigational gene therapy, and emicizumab (Helimbra) for hemophilia.