Number of orphan diseases

Portrait of Dr. Stephen GroftGroft’s journey, which includes many major milestones, started on a personal note. "Growing up in the 1950s, I had friends and neighbors who were stricken with diseases that had few or no effective treatments, " Groft said. "Some of the diseases of our time that I witnessed firsthand included cystic fibrosis, leukemia, brain tumors, Marfan syndrome, muscular dystrophy, cerebral palsy, polio, multiple sclerosis and Parkinson’s disease."

These personal connections led him to a career at the Food and Drug Administration (FDA) and NIH, where he worked to prioritize rare diseases research and orphan product development. Along the way, he also served in a variety of advisory roles to national leaders, including congressional and White House representatives, allowing him to influence the national agenda on rare diseases research.

Stephen Groft and SSA Commissioner"The creation of NCATS is in many ways a validation of Steve’s work over the past 30 years, " said NCATS Director Christopher P. Austin, M.D. "Rare diseases are no longer a curiosity on the periphery of the biomedical research enterprise. They now are central to the research agenda, and that is due in large part to Steve’s vision, dedication and effectiveness." Rare disease research is a crucial priority for NCATS. The Center fosters collaborative efforts with multidisciplinary research teams to develop new approaches to drug discovery for rare diseases.

The Early Years

"Dr. Groft is an exceptional individual whose passion and commitment to patients was unprecedented. He has led the rare diseases community by setting an example as to how we should work together in order to be successful and make a difference, and he practiced what he preached."
— Nicole Boice, president/founder, Global Genes | RARE Project

Groft began his career in 1968 as a small-town pharmacist in Pennsylvania, helping patients understand their conditions and medications and building connections that would influence his lifelong focus on rare diseases. His policy-related efforts began in 1982 at the FDA Office of Orphan Products, a division dedicated to advancing the evaluation and development of therapeutics for the diagnosis and treatment of rare diseases.

Popular Q&A

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Does the center for disease control and prevention conduct research in epidemiology?

The CDC has an Epidemiology Elective Program for students in their senior year studying medicine or to be a Veterinarian. Contact the CDC at (888) 232-6348. They are located at: 1600 Clifton Rd.Mailstop E-92 Atlanta, GA 30333. Good luck and !

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