Rare disease is defined in the United States as a disease that affects fewer than 200,000 people. There are over 7,000 current rare diseases affecting around 25 million Americans. Sometimes, these diseases affect as few as 12 people. While clinicians, scientists and pharmacists are taught about rare diseases during their undergraduate studies, few schools offer in depth assessment of the rare disease at the post doc or graduate level.
Fortunately, universities and medical schools are implementing more and more rare disease graduate and fellowship programs to identify and treat rare and debilitating diseases. Some of those programs are listed below.
Located in Claremont, CA, the Center for Rare Disease Therapies functions as a nonprofit think tank partnering with the pharmaceutical and biotech industry, government agencies and patient advocacy groups.
Teams of four to five students work with an industry liaison and a faculty mentor to complete their Team Masters Project-the capstone of KGI's Master of Bioscience program-by examining the technical, regulatory and business aspects of rare diseases.
The Rare Brain Disorders Program at UT Southwestern includes a patient care resource and a research laboratory devoted to the understanding of infrequent, undiagnosed, or underdiagnosed neurological diseases of children and adults.
The program partners with the Child Brain Foundation to hold a yearly conference.
Genzyme Fellowship Program for Global Commercial Strategy for Rare Diseases at the MCPHS University
A 2-year clinical documentation program helps students gain insight into the orphan drug development process as well as assist in providing commercial assessments for new and existing programs within the rare disease space.
The university also offers the Global Medical Affairs program where students can gain an understanding of rare genetic conditions, approved treatments and products in development while working cross-functionally as an integral member of the Global Medical Affairs team in a rotation-based experience.
The program allows students hands-on access to direct patient care. Students participate in teaching rounds and conferences with physicians representing rare disease medicine.
Students are assigned a resident who provides instruction on patient care duties. Students learn clinical decision making skills, assist in the operating room and perform physical examinations.
Postdoctoral pharmacy students can work to improve the care of patients with rare diseases through research on new drug therapies and education of health policy relating to rare diseases and orphan drugs.
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