TOPIC: Featured News, Patients & Members
Two weeks ago, as my flight landed in Boston, I stepped outside of the plane with a long tube in my arm, inside of which was a very special poster. It was the poster about a project I had completed with NORD. I was about to present this poster at the 2012 National Society of Genetic Counselors’ (NSGC) Annual… Read More
TOPIC: Featured News, Medical, Research, Industry
According to a report on the BBC News web site today, the European Commission has — for the very first time anywhere in the Western world — given final approval to market a gene therapy that corrects an inborn error of metabolism for the treatment of any type of human genetic disorder. Read More
TOPIC: Medical, Patients & Members, Research
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: (more…)
