The work of a CHOC metabolic disorders specialist and is highlighted in a recent Science magazine article.
The article hinges on Dr. Raymond Wang’s work to help a patient with a rare condition called Niemann-Pic Type C, a condition that causes cholesterol to accumulate in the brain, lungs, liver and spleen, leading to deterioration and early death.
Not only is the young girl doing well after Dr. Wang began an experimental treatment, but her family’s foundation also provided funding that allowed Dr. Wang to continue research that could help children with a rare metabolic diseases called mucopolysaccharidoses, or MPS.
To that end, the piece examines the tug of war felt by some physicians when balancing patient care and research. Despite a physician’s strong interest and commitment, research eludes many due to a lack of time and funding.
Read the full article in Science magazine.