Reena Kartha, PhD, Associate Director of Translational Pharmacology at UMN’s Center of Orphan Drug Research (CODR), discusses current research being done on Gaucher disease.
Gaucher disease is a rare lysosomal storage disorder in which glucocerebroside accumulates in cells and certain organs. The disorder is characterized by bruising, fatigue, anemia, low blood platelet count and enlargement of the liver and spleen.
As Dr. Kartha explains, there has been a lot of research lately regarding the large variability in treatment response for Gaucher patients. Notably, she acknowledges Dr. Sidranski and her research on potential small molecule chaperones for the treatment of gaucher. Dr. Kartha also acknowledges Sidhee Sahasrabudhe, a PhD student who presented her research on potential drug interactions between an oral Gaucher disease treatment and an investigational COVID-19 therapy at this year’s WORLDSymposium conference.
For more information about Gaucher disease and other lysosomal storage disorders, visit checkrare.com/diseases/lysosomal-storage-disorders/
