by Peter Ciszewski | Apr 25, 2019
Debra Miller is the founder and CEO of CureDuchenne, and in this video she provides an overview of the organization. Debra founded CureDuchenne in 2003 with her husband after their only son was diagnosed with Duchenne. The funds CureDuchenne raise support...
by Peter Ciszewski | Apr 24, 2019
Arndt Rolfs, MD, Chief Executive Officer of Centogene, discusses the challenges of diagnosing rare diseases. Dr. Rolfs tells the story of his early days of practicing medicine. While on vacation, he came across an article in the newspaper on a disease he had...
by Peter Ciszewski | Apr 23, 2019
Jeff Ajer, Executive Vice President and Chief Commercial Officer of BioMarin, discusses gene therapy. The development of these transformative therapies has triggered discussions about market access challenges, the viability of alternative financing mechanisms,...
by Peter Ciszewski | Apr 22, 2019
Peter Saltonstall, President and Chief Executive Officer of the National Organization for Rare Disorders (NORD) discusses some of the challenges facing the rare disease community, including the perception that drug pricing for orphan drugs is a driving force...
by Peter Ciszewski | Apr 18, 2019
The Food and Drug Administration (FDA) granted fast track designation to APR-246 for the treatment of patients with myelodysplastic syndrome who have TP53 mutations. In addition, FDA also granted Orphan Drug Designation to APR-246 for treatment of MDS. “The...
