Rare Disease Report

NORD Breakthrough Summit 2015

Richard Peters of Genzyme talks about the panel discussion he chaired during the NORD summit about promoting early diagnosis of rare diseases through physician education and awareness.
We talked with Wendy White, president of the Healthcare Businesswomen's Association about the organization and its goals.
Wendy White, SVP at Dohmen Life Science Services talks about the company's patient care model that is designed to help smaller patient populations such as those found in the rare disease community.
Zsa Zsa Stallings of Cure in Sight describes her organization and its goals to help patients coping with a diagnosis of ocular melanoma.
At most scientific or medical conferences, you will often see 1 or 2 people wearing a black jacket with a portrait on their back. They are part of the 'Walking Gallery'.
Richard Peters, MD, PhD, head of the global rare disease business unit at Genzyme talks about the importance of educating payors about rare diseases and orphan drugs.
Bert Bruce at Pfizer talks about some of the programs that his company has in development, including drugs to treat Duchenne muscular dystrophy, sickle cell disease, and hemophilia B.
At the NORD Rare Diseases and Orphan Products BreakThrough Summit, we talked with Susan Kahn, executive director of the National Tay-Sachs & Allied Diseases Association (NTSAD)
Janet Woodcock, MD, of CDER/FDA talks about the FDA's position on using biomarkers in pivotal clinical trials.
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