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Open minds
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An Unusual Diagnosis made, But How do You Treat it?
Many physician-scientists strive to address this through applied research. In this age of genetics and genomics we in Lebanon have to pull our own weight and contribute to medical and scientific progress. Support for research by our visionary OpenMinds board, made possible by the generosity of our donors has made this a reality. Work on the novel treatment, galactosylceramide, specific for the rare genetic disorder, CLN3 disease, has been licensed to SENEB, Inc. PhD student Sally El-Sitt had her work on a CLN3 mouse model presented both in Boston at the international NCL meeting and in Hamburg Germany at the NCL-Stiftung in Hamburg.Many physician-scientists strive to address this through applied research. In this age of genetics and genomics we in Lebanon have to pull our own weight and contribute to medical and scientific progress. Support for research by our visionary OpenMinds board, made possible by the generosity of our donors has made this a reality. Work on the novel treatment, galactosylceramide, specific for the rare genetic disorder, CLN3 disease, has been licensed to SENEB, Inc. PhD student Sally El-Sitt had her work on a CLN3 mouse model presented both in Boston at the international NCL meeting and at the NCL-Stiftung in Hamburg.
Three novel compounds tested on cell lines derived from kids with the deadly neurogenetic disorders CLN1/2/3/5/6/8 showed effectiveness in preventing cell death and lowering the levels of toxic ceramide in them. CLN2 and CLN6 disease occur in Lebanon. The ASKC clinic gets numerous referrals of kids with CLN3, CLN2 and CLN6 disease from Syria, Iraq, Kuwait and the Ukraine.
Fadi Saadeh, an AUBMC 4th year medical student presented this work at the International NCL conference in Boston, and received a $ 1000 award from the Diana Tamari Sabbagh foundation for best scientific research for 2017. Based on this work a new patent application has just been submitted entitled: Functionalized Pyridine Carbamates with Enhanced Neuroprotective Activity. Inventors: Paul Trippier, Nihar Kinarivala, Rose-Mary Boustany (Texas Tech University and AUB). Application number 62/532,624. Filing date: 7/14/2017.