Latest Results from the Morquio A Registry Study (MARS)

  Barbara Burton, MD of Lurie Children’s Hospital of Chicago discusses the latest results from the Morquio A Registry Study (MARS). The registry data shows that persons with Morquio syndrome type A who are taking enzyme replacement therapy (ERT) experience...

Castleman Disease Explained

  Ruth-Anne Langan Pai, PHD Candidate and the collaborative research project lead for the Castleman Disease Collaborative Network (CDCN) provides an overview of Castleman disease. Castleman disease is a group of rare disorders characterized by lymph node...

The Latest Ibrutinib Data for Treating CLL

  James Dean, MD, senior medical director at Pharmacyclics provides an update on the most recent long-term data showing ibrutinib to be safe and effective for treating patients with chronic lymphatic leukemia (CLL). CLL is a rare blood cancer resulting in a...

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...

WORLDSymposium 2020 Preview

On February 10 – 13, 2020, leading lysosomal disease researchers will come together to share the latest scientific information about rare lysosomal conditions at WORLDSymposium. The annual event provides an opportunity for basic, transitional, and clinical researchers...

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...
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