Scientists and Clinicians Unite to Fight Rare Childhood Disorder

The Legacy of Angels Foundations sponsors the 2nd Krabbe's Translational Research Network Program for the study of neurodevelopment in rare disorders.

Pittsburgh, PA – for two and one half days the leading scientists researching the rare childhood disease, Krabbe's met with practitioners and other experts to bring the latest research into the clinic where it can improve the outcome for children with Krabbe's Disease.Paul and Sue Rosenau, who created The Legacy of Angels Foundation after their family experienced the tragic death of a grandchild due to Krabbe's, sponsored the program.  The Rosenau’s have stated, 

We wanted to focus our philanthropy on the education and awareness of this disease and help fund research for better treatments and ultimately a cure for Krabbe's disease.

Krabbe's disease is a rare, inherited degenerative disorder of the central and peripheral nervous systems.  It is characterized by the presence of globoid cells (cells that have more than one nucleus), the breakdown of the nerve’s protective myelin coating, and destruction of brain cells.  The early-infantile form of this disease affects infants, with onset before age 6 months. Without early diagnosis and treatment Infantile Krabbe's disease is generally fatal before age 2

As part of their effort to fight this disease The Legacy of Angels Foundationsponsored the 2nd annual Krabbe's Translational Research Network meeting, held March 12- 14 at the Hilton Garden Inn in Pittsburgh, PA.

Maria L. Escolar, M.D. who directs The Program for the Study of Neurodevelopment in Rare Disorders (NDRD) at the University of Pittsburgh Medical Center and has created a Virtual Medical Home and Clinic for Children with Krabbe's Disease located on the web at: http://ndrdvirtualmed.com/index.shtml hosted the meeting.  More than twenty doctors, researchers, clinicians, and family members participated.

Topics addressed included “New Tools and Advances in Treating,” “Factors Inducing Disease Progression,” “Stem Cell Therapies,” “Novel Gene Therapy Approaches to the Treatment of Krabbe's,” “Challenges in Translating from Bench to Bedside,” and “Future Directions.”

Dr. Maria L. Escolar summed up the outcome of the meeting as, 

We have identified new projects that have potential to improve therapy; and developed teams of translational researchers that will collaborate towards our goals.