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November 2, 2012

TOPIC: Featured News, Medical, Research, Industry

Glybera Becomes First-ever Gene Therapy Approved in Europe

Posted at November 11, 2012 07:37 pm by

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

October 31, 2012

TOPIC: Medical, Patients & Members, Research

Creative Collaborations May Be Key to Reducing Chemotherapy Risks

Posted at October 10, 2012 01:33 pm by Marsha Lanes

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…)