The U.S. Food and Drug Administration (FDA) hosted a workshop on July 21, 2021 to discuss the disease characteristics, natural history, and endpoints to assess treatment benefit in patients with eosinophilic gastrointestinal disorders (EGID) beyond eosinophilic esophagitis (EoE).
Reena Kartha, PhD, MS, was named Rare Disease Difference Maker by Engage Health. Kartha is an assistant professor in the College of Pharmacy at the University of Minnesota and former fellow with the RDCRN’s Lysosomal Disease Network (LDN).
Glenn Furuta, MD, has received the North American Society for Pediatric Gastroenterology, Hepatology and Nutrition (NASPGHAN) 2021 Harry Shwachman Award. Furuta was recognized during the 2021 NASPGHAN Annual Meeting on Monday, December 13.
Dr. Rothenberg, the Principal Investigator for CEGIR, has been awarded the highest honor bestowed by the International Eosinophil Society for his major contributions to the advancement of our understanding of the eosinophil.
NEPTUNE has embraced the concept of Precision Medicine proposed by the Institute of Medicine in 2011. The Precision Medicine strategy proposes the development of new disease definitions generated by a multilayered analysis of the disease course while patients receive their usual clinical care in observational studies.
Through a recently awarded Clinical Trial Readiness grant from NINDS, CReATe investigators are now tapping into the invaluable resource that is the CReATe Biorepository in an effort to clinically validate leading biological fluid-based biomarker candidates to aid the design and implementation of future clinical trials.
The National Center for Advancing Translational Sciences (NCATS) at NIH is keenly interested in seeing more research studying rare diseases in which efforts and expertise are pooled across research groups and diseases to identify commonalities and shared targetable mechanisms. The intention is that such collaboration will increase efficiency and capacity in the development of new therapies compared to traditional approaches that focus within a single disease.
It is becoming increasingly clear that problems with some basic cellular processes can contribute to diseases affecting different organs. One cellular signaling pathway that appears to be involved in multiple diseases is the mTOR pathway.