Research Progress Report by Michael Sekar, PhD, AMMA Therapeutics, Inc.
In the spring of 2020, CRF awarded a grant to AMMA Therapeutics for $304,064, the aim of their study is to develop a once-daily subcutaneous
JOIN OUR JOURNEY TO A CURE ON RARE DISEASE DAY!
There are approximately 7,000 rare diseases that affect 30 million people in the U.S. and 300 million people in the world, and only 5% have
Dr. Cherqui Update from UCSD Stem Cell and Gene Therapy Clinical Trial!
CRF has a positive update from the UCSD Stem Cell and Gene Therapy clinical trial! This trial is possible because of the dedication and commitment
Final Research Progress Report Ming Li, PhD and Jacob Kitzman, PhD, University of Michigan
The research study, “Dissect the Protein Turnover Mechanism of Cystinosis Mutants” received a CRF research grant of $285,648 in 2018. Dr. Li explained in lay
Final Research Progress Report Sihoun Hahn, MD, PhD, Seattle Children’s Hospital Research Institute
CRF has supported Dr. Hahn’s important research since 2017, awarding $234,458 for his research on Newborn Screening. The aim of the study was to develop
Stem Cell Trial Update: One Year Later-Jordan Janz Shares His Thoughts
I definitely feel better today than when I had cystinosis. In the mornings I can go a couple of hours or so without eating and