Collaboration Yields Possible Treatment for Rare Neurodegenerative Disorder
A discovery in bacteria and a hallway conversation led St. Jude Children’s Research Hospital scientists to a potential therapy for a debilitating childhood neurodegenerative disorder
St. Jude Children’s Research Hospital investigators have developed a new class of compounds that extended the lives and eased symptoms of mice with a progressive neurodegenerative human disorder.
The rare, inherited disorder, pantothenate kinase-associated neurodegeneration (PKAN), affects up to three in a million individuals and is caused by mutations in the PANK2 gene. Iron accumulates in brain cells, and patients have difficulty walking, swallowing, chewing and performing other activities. There is no treatment.
PKAN is thought to occur when neurons lack an adequate supply of the molecule coenzyme A (CoA), preventing them from functioning normally. PANK2 is one of three closely related proteins (isoforms) that regulate CoA production.
Click here to read more.
Microsurgical and Radiological Anatomy of Cerebral Sulci, Gyri, and Ventricles: The Rhoton-de Oliveira Course for Surgical Applications
Nov. 13-15, 2019; Jacksonville, Fla.
Complex Endoscopic Endonasal Surgery of the Skull Base
Nov. 14-16, 2019; Pittsburgh
2019 New Frontiers in the Diagnosis and Management of Movement Disorders
Nov. 16, 2019; Chicago
9th World Congress of Neuroendoscopy
Nov. 21-24, 2019; Orlando, Fla.
Mayo Clinic Multidisciplinary Spine Care Conference 2019
Nov. 22-23, 2019; Amelia Island, Fla.