Funded Research Projects & Initiatives:

Clinical Trial Implementation Fund:  2024-Present

In July 2024 the FDA officially approved the FIRST Human Clinical Trial for THREE HNRNPH2-RNDD patients!  n-Lorem Foundation has worked over the last few years to develop the ASO drug and provides the drug itself at no cost.  The Clinical Trial is being administered at Columbia University Irving Medical Center.  But that is not the end!  It is up to US to raise the overhead costs for administering the clinical trial. YBRP has raised $150,000 toward the Clinical Trial Implementation Fund to ensure the success of this program and bring us closer to broader clinical trials and treatment for ALL those with HNRNPH2-RNDD.

Natural History Study (NHS): 2018-Present

YBRP has provided approximately $110,000 in funding towards the NHS. Dr. Bain and her team are actively recruiting HNRNPH2 patients for the crucial Natural History Study.  The greater the number of participants, the deeper the understanding of the disorder.  This study is fundamental to current and future research into potential treatments and/or a cure and is key in developing a "road map" and plan of care for doctors, clinicians, parents, and care-givers to be able to give the proper care to a patient throughout the course of their development.