ResearchFocus on the pharmaceutical and biotech industry
Amrita Krishnan, MD, hematologist-oncologist at the City of Hope Comprehensive Cancer Center talks about what the future holds for managing people with multiple myeloma.
Claudio Santos, MD, MBA of PTC Therapeutics provides an update on the orphan drug being developed by the company, in partnership with Roche, to treat spinal muscular atrophy (SMA).
Adrian Woolfson, MD, PhD, of Sangamo Therapeutics talks about the physical and psychological advantages of using gene therapy to treat hemophilia A.
Gallia Levy, MD, PhD, Associate Group Medical Director, at Genentech explains why joint pain is so prevalent in persons with Hemophilia A.
Rajiv Ratan, MD, PhD, of the Burke Neurological Institute, explains the mechanisms involved in how cells undergo ‘programmed death’ following a hemorrhagic stroke.
Gaurav Shah, MD, Chief Executive Officer & President of Rocket Pharmaceuticals explains the advantages that smaller biotech companies have in developing treatments for rare diseases.
David Pearce, PhD, of the Coordination of Rare Diseases at Sanford (CoRDS) talks about Sanford Health, one of the largest health systems in the United States.
Peter Voorhees, MD, of the Levine Cancer Institute in Charlotte NC discusses the emerging treatments for multiple myeloma that are focused on BCMA-targeted therapies.
Snejana Krassova, MD of Bayer Pharmaceuticals talks about the need for more treatment options to be available for persons with hemophilia A.
Claudio Santos, MD, MBA, of PTC Therapeutics describe the pathophysiology of Aromatic l-amino acid decarboxylase (AADC) deficiency and how it is diagnosed. AADC deficiency is a rare genetic disorder due to mutations in the DDC gene that regulates the AADC...