My friend’s 18-year-old son died recently following a 6-month battle with acute myeloid leukemia. There are many different stories I could tell about Tucker’s experience that are all too familiar to those in the rare disease community: read more >
2011 and 2012 have been good years but the rate of innovation in drug development is anything but healthy, a venture investor focusing on the life sciences said yesterday at the U.S. Conference on Rare Diseases and Orphan Products underway this week in Washington DC. read more >
The regulatory pathway that we have learned from rare diseases is now becoming the paradigm in general, FDA Deputy Commissioner Stephen P. Spielberg, MD, PhD, said this morning in the opening session of Day 2 of the U.S. Conference on Rare Diseases and Orphan Products.
In an extraordinary signal of the progress that is being made to find treatments for some types of rare disorders, an advisory committee to the U.S. Food and Drug Administration (FDA) has recommended approval this week for not just one but two new drugs to treat homozygous familial hypercholesterolemia (HoFH).