Headshots of Donald Kohn, MD, and Jolan Walter, MD, PhD
January 24, 2022

The SCID, Angels for Life Foundation, a patient advocacy group of the Primary Immune Deficiency Treatment Consortium (PIDTC), has announced the winners of its 2022 Grant Awards.

Research Roundup. #RareDiseasesSpotlight. Rare Diseases Clinical Research Network
January 21, 2022

Each month, we share summaries of recent Rare Diseases Clinical Research Network (RDCRN) grant-funded publications. Catch up on the latest RDCRN research below.

Headshot of Danielle Arnold, MD
January 19, 2022

Danielle Arnold, MD, is an attending transplant physician at the National Institutes of Health. She is also a member of the Primary Immune Deficiency Treatment Consortium (PIDTC). Her research focuses on improving transplant outcomes for patients with primary immune deficiencies and immune regulatory disorders. Here, she shares her start in rare disease research, exciting discoveries, and future goals.

Marc Rothenberg, MD, PhD, with a future researcher of eosinophilic disease
January 13, 2022

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). 

Headshot of Reena Kartha, PhD, MS
January 10, 2022

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).

Mitchell Magyar with his mother and sister, wearing sunglasses and smiling
December 16, 2021

Mitchell Magyar is a 13-year-old who loves golf, hiking, archery, riding his bike, and playing video games with his friends. He is also diagnosed with argininosuccinic aciduria (ASA), a type of urea cycle disorder. Here, his mother Amy Magyar shares their journey with ASA, from diagnosis to getting involved with advocacy and research participation.

Headshot of Glenn Furuta, MD
December 14, 2021

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.