What is a Rare Cancer?

  Eric T. Wong, MD, Associate Professor at Harvard Medical School provides an overview of rare cancers. Unlike rare diseases that are defined as affecting less than 200,000 people in the United States, rare cancers are referred to those affecting less than 45,000...

Using Social Media Data to Improve Drug Development

  Maria Picone is the mother of a child with Prader-Willi syndrome as well as founder/CEO of TREND Community – a company that collects anecdotal information from patients and caregivers so that the information can be quantified and assessed more efficiently. The...

Lipodystrophy Explained

  Andra Stratton, president of Lipodystrophy United provides an overview of lipodystrophy. Lipodystrophy is a rare medical condition in which persons have an abnormal distribution of fat in the body. That abnormality can present itself in many ways. People with...

Recruiting Patients for Rare Disease Clinical Trials

  Sandy Macrae, PhD, Chief Executive Officer of Sangamo Therapeutics, a company developing genetic therapies for a variety of rare conditions, including hemophilia A, Fabry disease, beta-thalassemia, sickle cell disease, mucopolysaccharidosis I (MPS I) and MPS...

RUSP and the ScreenPlus Panel

  Melissa Wasserstein, MD, Chief of Pediatric Genetic Medicine at the Children’s Hospital at Montefiore, talks about Recommended Uniform Screening Panel (RUSP). RUSP is the list of diseases, mostly rare diseases, that the Secretary of the Department of Health and...
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