NIH awards Penn Medicine and Children’s Hospital of Philadelphia $26 million grant to develop therapies for rare newborn genetic diseases
A Penn Medicine and Children’s Hospital of Philadelphia team will seek to develop treatments for three rare, incurable genetic diseases that impact newborns in the first weeks and months after birth: Phenylketonuria (PKU), hereditary tyrosinemia type 1 (HT1), and mucopolysaccharidosis type 1 (MPSI), commonly known as Hurler’s Syndrome.
FULL STORY AT Penn Medicine News →