BEIRUT: The American University of Beirut announced Thursday the invention of a new drug to treat CLN3 gene disease, which is considered one of the most dangerous diseases in children.
The CLN3 gene maintains the integrity of the body’s cells. The failure of this gene leads to the death of brain cells and the relentless clinical and neurological deterioration leading To Batten disease with blindness, inability to talk, acute seizures, paralysis and early death.
The invention of the drug – Galactosylceramide – was the result of intensive efforts by the AUBMC’s chief of Pediatric Neurology Division and Director of Neurogenetics Program and Special Kids Clinic Rose-Mary Boustany and her team.
“It is hard not to be able to give an anxious family an accurate diagnosis for their child. It is harder to make a diagnosis and tell the family there is no treatment since it is every physician-scientist’s hope to develop a cure for the ills that afflict their patients,” Boustany said while adding that scientist have to partner up with the pharmacological industry to move discoveries into the clinic and market place.
Rose-Mary Boustany, MD, is Professor of Pediatrics and Adolescent Medicine and Professor of Biochemistry and Molecular Genetics. She is also a Duke Adjunct Professor of Pediatrics.
