by Peter Ciszewski | Dec 22, 2018
Richard A. Basile Co Founder and CEO of BioPontis Alliance for Rare Diseases, discusses his organization’s business model and focus on rare diseases. BioPontis is an international nonprofit organization whose mission is to advance promising science into...
by Peter Ciszewski | Dec 21, 2018
Annette Bakker, PhD, President and Chief Scientific Officer, of the Children’s Tumor Foundation discusses neurofibromatosis, also known as NF, is a genetic disorder that affects 1 in every 3,000 people. There are three types of NF: NF1, NF2, and...
by Peter Ciszewski | Dec 20, 2018
At the 2018 American Society of Hematology (ASH) Annual Meeting in San Diego, Dr. Howard Levy, Chief Medical Officer of Catalyst Biosciences discusses hemophilia and his company’s poster at the meeting: Updated Positive Interim Data from Its Phase 2/3...
by Peter Ciszewski | Dec 19, 2018
Heather A. Lau, MD, Director, Lysosomal Storage Disease Program at NYU Langone in New York City discusses Mucopolysaccharidosis type III (MPS III), also known as Sanfilippo syndrome. This progressive disorder primarily affects the brain and spinal cord (central...
by Peter Ciszewski | Dec 18, 2018
The FDA approved romiplostim for treatment of pediatric patients with immune thrombocytopenia. The approval of romiplostim (Nplate, Amgen) applies to children aged 1 year or older who have had immune thrombocytopenia (ITP) for at least 6 months and had insufficient...
