The Duchenne muscular dystrophy community is facing its most monumental decision in the history of the disease and May 26th we are asking the FDA to approve the first safe and effective treatment called ETEPLIRSEN. Duchenne Muscular dystrophy is one of the MOST DEADLY and most common genetic disorders which affects thousands of boys world wide. THERE ARE NO APPROVED THERAPIES Thousand of boys and families world wide are waiting for the FDA to VOTE YES TO APPROVE ETEPLIRSEN. On April 25th Advocates for Duchenne, boys afflicted with the disease from all over the world, parents, members of government, doctors, and scientist went to Washington DC to petition the FDA to approve this drug. Unfortunately the advisory panel narrowly rejected it despite sound scientific evidence of efficacy, pleas from numerous experts, families, and the boys themselves. THE FDA HOWEVER CAN OVERTURN THIS DECISION BY MAY 26th. Without this drug thousands of boys will be condemned to wheelchairs in there teens and death in their 20s. It is imperative the drug ETEPLIRSEN by Sarepta Therapeutics be approved by FDA.  Boys with Duchenne Muscular Dystrophy (DMD) are born without the dystrophen Protein (A protein in muscle to absorb shock) , and without it their muscles degrade and these boys end up in wheelchairs by their teens and die in their 20s. The drug Eteplirsen acts to partially restore the production of this dystrophen protein which has been shown to stabilize children in their teens who have been on it for 4 years. Nearly 100% of the boys on the drug are now making a small amount of this dystrophen protein.  THIS NEW PROTEIN THE BOYS ARE MAKING WITH THE HELP OF THIS DRUG appears to have stabilized their walking ability for the past 4 years which is FAR DIFFERENT than boys who have not gotten the drug. 10/12 boys are still walking where only 2/12 control group patients without the drug would be expected to walk after 4 years. Not only has the scientific data (muscle biopsies have shown the new protein, and walk test have shown kids on drug to walk further than those off of it) been positive but the mothers and families have reported great improvements in their childrens day to day abilities. See below link for data on Eteplirsen.  http://phx.corporate-ir.net/External.File?t=1&item=VHlwZT0yfFBhcmVudElEPTUyMDQyNzd8Q2hpbGRJRD01OTYxNDg=

We in the DMD community are ASKING THE PUBLIC TO SHOW THEIR SUPPORT FOR THE BOYS AFFLICTED WITH DUCHENNE by INSISTING TO CONGRESS and to the WHITE HOUSE that the FDA APPROVE THIS FIRST EVER THERAPY FOR THIS FATAL ILLNESS.  THERE ARE NO OTHER DRUGS TO TREAT THESE CHILDREN!!!   To read more about the stories of these children and for ways to help please visit  http://jettfoundation.org.  

WE ASK FOR YOUR HELP TO SIGN THIS PETITION and  we ask that the FDA MAKE HISTORY BY APPROVING THE FIRST EVER THERAPY for DUCHENNE on May 26th 2016. 

JOIN US IN ASKING FOR APPROVAL SO THESE KIDS CAN HAVE RENEWED HOPES OF A BRIGHTER AND HEALTHIER FUTURE !!!!