rare disease day 2019

12th Annual Rare Disease Day Raises Awareness for Therapies

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Keck Graduate Institute (KGI) hosted its 12th Annual Rare Disease Day on Friday, March 1 with presentations from industry leaders, a poster session from KGI students, and a special film screening followed by a question-and-answer session.

The event served as an opportunity for students, faculty, and guests to collaborate, discuss, and raise awareness for rare disease therapies. Attendees were officially welcomed by KGI President Sheldon Schuster, who also serves as vice chairman for the National Organization for Rare Disorders (NORD).

Prior to the official start of the program, President Schuster and members of KGI’s Rare Disease Club honored Ian Phillips, KGI Norris Professor of Applied Life Sciences, for his founding and continuous support of Rare Disease Day and the student club on campus.

“Dr. Phillips’ passion for rare disease advocacy, innovative science, and ethics have inspired a whole new generation of healthcare providers,” said Jennifer Nguyen, president of KGI’s Rare Disease Club.

KGI alumnus Tom Lester, MBS ’04, kicked off the presentation portion with a discussion on leading the development of rare disease therapies by journeying together. Lester, the senior director for product development at BioMarin Pharmaceutical Inc., shared stories from families directly affected by rare diseases, and offered advice to KGI students about working within the field.

“There are a few takeaways that I want to convey to students: the first is that patients are experts by experience, and patient families put a lot of trust and faith in scientists,” said Lester. “You have to be prepared for tough questions and to answer them honestly; take the time to explain evidence in a way that is understood by all; don’t let perfection be the enemy of ‘good enough’; and be sure to align your core values with those of the patients you aim to treat.”

Kathryn Moseley, assistant professor of pediatrics at the USC Keck School of Medicine, spoke about the rare disease Phenylketonuria, shared her personal story of the disease, and provided research about best practices for treating patients. Lorraine Dressler, advocate for the Myotonic Dystrophy Foundation, spoke about living with the rare disease and how it affects her family.

The event continued with a film screening of Special Blood, a movie about four patients with a life-threatening rare disease who find strength in each other and their community. The screening was followed by a Q&A with Jessica Cummings, senior technical communicator at Takeda Pharmaceutical Company, and Rana Alhawi, associate director for manufacturing support at Takeda.

KGI President Sheldon Schuster honoring Ian Phillips