Gene therapy offers long-awaited hope for children with rare, incurable disorder

Children with a devastating genetic disorder characterized by severe motor disability and developmental delay have experienced sometimes dramatic improvements in a gene therapy trial launched at UCSF Benioff Children’s Hospitals. The trial includes seven children aged 4 to 9 born with deficiency of AADC, an enzyme involved in the synthesis of neurotransmitters, particularly dopamine, that…

Magnetic field from MRI affects focused-ultrasound-mediated blood-brain barrier

IMAGE: In a mouse model study of MRI-guided focused ultrasound-induced blood-brain barrier (BBB) opening at MRI field strengths ranging from ­approximately 0 T (outside the magnetic field) to 4.7 T, the… view more  Credit: Washington University in St. Louis MRI-guided focused ultrasound combined with microbubbles can open the blood-brain barrier (BBB) and allow therapeutic drugs to…

Do more visits with kidney specialists improve dialysis patient-reported outcomes?

Highlights Patients with kidney failure did not report better experience with care from more frequent face-to-face visits with kidney specialists at dialysis facilities. In fact, more frequent visits were linked with slightly lower patient-reported experiences with kidney-related care. Washington, DC (July 12, 2021) — In a recent analysis, more frequent kidney specialists’ visits to clinics…