Having 20 years’ experience with biotech companies, Raj Prabhakar serves as a Chief Business Officer and Senior Vice President of Rocket Pharmaceuticals in New York City. Raj Prabhakar has a range of professional interests, notably rare pediatric diseases. One of the least common of these illnesses is progeria, also known as Hutchinson-Guilford syndrome. Children with the condition age prematurely during their first two years of life, leading to hardening of the arteries, stroke, and other cardiac problems. Their average life expectancy is about 13 years. Progeria features a range of symptoms, such as below normal height and weight, an unusually large head compared to the face, and a high-pitched voice. Other signs include large eyes that don’t fully close, thin and wrinkled skin, prominent veins, and early hair loss. Patients experience stiff joints, reduced hearing, weak bones, and immature teeth, among other internal issues. The disease is extremely rare - at any one time only 400 or so of the world’s population have it. Progeria is caused by a single-gene mutation that possibly inhibits the creation of enzymes that slow the aging process. Progeria is incurable, but a new treatment has just reached the market. The medication, called Zokinvy, has been shown in clinical trials to enhance patients’ blood flexibility and improve their weight, hearing, and bone density.
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