Minneapolis, MN, November 30th, 2023 — KrabbeConnect, a pioneering organization dedicated to supporting individuals and families affected by Krabbe disease, is proud to announce the receipt of an important grant from the Rosenau Family Research Foundation (RFRF). This grant, titled “Re: Parent-Reported Disease Burden in Infantile and Late Infantile Krabbe Disease; A Comparison of Quality-of-Life Outcomes in Transplant and Non-or-Late Transplant Patients,” will spearhead pivotal research in understanding and enhancing the quality of life for those battling Krabbe disease.
Krabbe disease, a rare and devastating genetic disorder, affects the The nervous system of the body is made up of the Central Nervous System (CNS) and the Peripheral Nervous System (PNS)., primarily in infants, leading to severe neurological complications. KrabbeConnect has been at the forefront of advocating for research, support, and awareness for this condition, aiming to improve outcomes and care for affected individuals and their families.
The generous funding from the Rosenau Family Research Foundation will facilitate a comprehensive study examining the disease burden experienced by both infantile and late infantile Krabbe disease patients. This 2-year study aims to delve into the quality-of-life outcomes, particularly comparing patients who have undergone transplantation and those who have not or had delayed transplants.
“We are immensely grateful to the Rosenau Family Research Foundation for their unwavering support and commitment to advancing research in the realm of Krabbe disease,” said Stacy Pike-Langenfeld, President of KrabbeConnect. “This grant will enable us to conduct crucial research that can significantly impact the lives of those affected by this rare condition.”
This study will be led by Dr. Nicholas Bascou. Dr. Bascou completed his undergraduate studies at the University of Pittsburgh at The Program for the Study of Neurodevelopment in Rare Disorders (NDRD). He then went on to obtain his M.D. at the Cooper Medical School of Rowan University in Camden, New Jersey and completed his training in internal medicine at Albert Einstein Medical Center in Philadelphia, PA. Dr. Bascou, currently a physician resident in Neurology at Johns Hopkins University states, “It is incredible to see the leaps in our understanding of Krabbe disease over the past decade and the new treatments that are becoming available. Our team is extremely grateful to RFRF for providing the resources needed to help conduct a study that we hope will demonstrate how big of an impact NBS and disease modifying therapies have on the lives of patients and their families affected by Krabbe.”
The study intends to gather valuable insights through parent-reported data, enabling a comprehensive assessment of the challenges faced by individuals living with Krabbe disease. By comparing the quality of life among patients who have received transplants and those who haven’t, the research aims to shed light on potential differences in outcomes, informing future treatment strategies, patient care, and most importantly, the importance of early intervention.
The funding received from the Rosenau Family Research Foundation reaffirms the commitment of both organizations to catalyze advancements in Krabbe disease research and ultimately improve the lives of patients and their families worldwide.
KrabbeConnect is a non-profit organization dedicated to supporting individuals and families affected by Krabbe disease. Through advocacy, education, and research initiatives, KrabbeConnect aims to improve the quality of life for those impacted by this rare genetic disorder. To learn more about KrabbeConnect and its initiatives, please visit https://krabbeconnect.org/
About Rosenau Family Research Foundation:
Rosenau Family Research Foundation (RFRF) is a private non-profit organization dedicated to improving the lives of patients impacted by Krabbe disease and Cystic Fibrosis through research funding and disease advocacy, and helping individuals live life undefined by Krabbe disease and Cystic Fibrosis. To learn more about RFRF, please visit https://rosenaufoundation.org/our-story