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November 21, 2012

TOPIC: Patients & Members

Barretstown – a Serious Fun Camp!

Posted by Lisa Sencen

When I arrived through the gates at the Barretstown camp, I was eager to meet my campers and fellow group of volunteers, caras, Gaelic for friend. What I didn’t know is what a lasting impact this camp, set in Ballymore Eustace, Ireland, would have on my life. (more…)

November 12, 2012

TOPIC: Featured News, Patients & Members

The Story Behind the Rare Disease Day School Curriculum

Posted by

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

November 2, 2012

TOPIC: Featured News, Medical, Research, Industry

Glybera Becomes First-ever Gene Therapy Approved in Europe

Posted 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 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…)