dr. beall, you talked about the development of mutation specific therapies and the next evolution in precision medicine. and you can see the cystic fibrosis example impacting the way we address other serious illnesses. is there something more you would like to add to that. >> we are in the age of personalized medicine. fortunately with the completion of the human genome. we understand the genetic diseases it is a critical time for us. dr. collins downstairs, he is excited because he was one of the discoverers of our cf gene. we live in a unique age. more and more therapies are going to be developed towards specific mutations. that's why we have to have patient registries. when they felt they had a drug that could work on one mutation we were able to tell them in the united states we have 1100 of those patients within five minutes after they asked us because of a patient registry because we have a documented history of the disease. i think that's why it is important to have personalized medicine

beall, president and ceo of the cystic fibrosis foundation. third, esther richard pops, chairman and ceo of alkermes. fourthly, dr.ncer society. finally, dr. marshall summar, director, scientific advisory committee national organization for rare disorders. thank you all for coming. we'll each have five minutes to summarize your testimony. your written testimony will be placed in the record. ms. furlong, we will start with you. you are recognized for five minutes for your opening statement. >> good morning, chairman pitts, and members of the committee. my name is pat furlong. 20 is ago i joined other pairs to form parent project muscular dystrophy two and duchenne from one of the many forms of martial distant and those common lethal this order back to solid. in 1984 i received the horrific diagnosis on my two sons, christopher and patrick, above of my sons are gone now. i waged his crusade in their honor. much has happened over the past 15 years to transform the duchenne landscape. and much of this is the direct result of the action by congress and this committee notably the enactment of the child health act in 200