ResearchFocus on the pharmaceutical and biotech industry
Barbara Burton, MD of Lurie Children’s Hospital of Chicago discusses the latest results from the Morquio A Registry Study (MARS).
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.
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).
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 people in this country.
On February 10 – 13, 2020, leading lysosomal disease researchers will come together to share the latest scientific information about rare lysosomal conditions at WORLDSymposium.
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.
Snejana Krassova, MD of Bayer Pharmaceuticals talks about the reality that some people have, or can develop, antibodies to the vectors that are commonly used for gene therapy.
Eric T. Wong, MD, Associate Professor at Harvard Medical School discusses some of the reasons that a patient will be interested in participating in a clinical trial, especially one involving a rare disease.
Claudio Santos, MD, MBA of PTC Therapeutics describes his company’s treatment options for Duchenne muscular dystrophy (DMD), including Translarna (ataluren) in Europe and Emflaza (defazacort) in the United States.
Gaurav Shah, MD, Chief Executive Officer & President of Rocket Pharmaceuticals explains Fanconi anemia, a rare blood disorder that his company is developing a gene therapy to treat.