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...

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...

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...

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...
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