FOR IMMEDIATE RELEASE              CONTACT:  Jen Dumm  (440) 570-2167
                                                                                                                   Jodi Nichols    (740) 502-6519
Two Ohio mom’s quest to change the world for their fatally ill sons
 
Jodi Nichols, Newark, Ohio and Jen Dumm, North Royalton, Ohio will testify at the Open Public Hearing at FDA Headquarters on January 22, 2016.  Both their sons, Andrew Nichols (9) and John Owen Dumm (12) are part of a 2 year drug trial at Nationwide Children’s Hospital in Columbus, Ohio.  The drug, Eteplirsen, has changed the course of the fatal illness the two boys share, Duchenne Muscular Dystrophy.  The mothers will join over 300 Duchenne families, all present to urge the FDA to say Yes to Eteplirsen.

Duchenne is a genetic muscle disease, which since birth, has deteriorated every muscle in these boys, to the point of wheelchair dependence and 24/7 care.  Once active, seemingly normal boys, Duchenne over time has robbed the simple things like, walking, brushing teeth and independence.  Duchenne is a brutal muscle killer that until Eteplirsen, would not be slowed or stopped.

Both boys are gaining strength while on this experimental drug.  Both are able to move arm, leg and trunk muscles that had been lost, which both mothers thought was permanent.  “Eteplirsen has dramatically slowed the course of this fatal illness in John Owen, he has strength in every muscle that we thought was gone for good.” Says Jen Dumm.

 The FDA is under fire by the rare disease community for recently denying the New Drug Application of Bio Marin’s drug, Kyndrisa, a similar drug as Eteplirsen.  This despite the urging of Congress to fast track such drugs through the FDA via the Food and Drug Safety and Innovation Act (FDASIA) passed in 2012.  

Eteplirsen is safe, both boys have shown zero negative side effects and the list of new found skills they can perform is endless.
 
These mothers beg for your support as they journey to Washington, DC on Friday 1/22/2016 to urge the FDA to pass this life changing drug produced by US Biotech Company, Sarepta Therapeutics.  Contact your congressman (click for House and click for Senate) and urge them to sign the Congressional Letter circulating on Capitol Hill in support of Duchenne Families.  Congress has given the FDA authority to approve drugs, such as Eteplirsen based on safety of the drug. 
 
“What would you do if your child was diagnosed with a killer of a disease and a treatment was within your reach, but it was up to the FDA to determine your child’s fate?”  Asks, Jodi Nichols.  “Please join Jen and I as we fight for our boys and urge your congressman to sign this letter encouraging the FDA to end our fight and the fight of many mothers across the country.”
 
About Duchenne:  Duchenne Muscular Dystrophy is 100% fatal and typically only affects boys.  A very tiny part of the boy’s X chromosome was deleted.  This deletion makes it impossible for him to build muscles because the message ‘build dystrophin’ never gets to his brain.  Eventually, all of the muscles in his body will breakdown making it impossible to walk, eat and breathe.  Soon after that his little heart muscles will breakdown as well and we will be forced to say goodbye to him well before he has fully enjoyed his 20’s. One in 3,500 boys has Duchenne Muscular Dystrophy.  Until Eteliprsen, there has been no effective treatment for Duchenne.
 
About Sarepta Therapeutics: Sarepta Therapeutics is a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare, infectious and other diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying Duchenne muscular dystrophy (DMD) drug candidates, including its lead DMD product candidate, eteplirsen, designed to skip exon 51. Sarepta is also developing therapeutics for the treatment infectious diseases, such as drug-resistant bacteria and other rare human diseases. For more information, please visit us at www.sarepta.com.
 
####

Dear Friends!  Happy New Year!  This update finds us in an urgent state of need.  As indicated in our last update, the FDA meeting is coming! 

Your action is needed BEFORE  this  THURSDAY - JAN 7 2016 - to send this message to the FDA regarding Eteplirsen!
 
NOW IS THE TIME!  Please stay with me on this long update and read it to the end – then ACT – WE MUST HAVE YOUR ACTION THIS WEEK…. It is Critical!

On January 22, 2016 the FDA will hold an open hearing on this drug.  This is our opportunity as our JOA community to have the FDA hear our voice and record our plea to approve this safe and effective drug…….

Are you in???  If so – Keep Reading then ACT!

We have seen amazing improvements in Owen since he started this medicine on April 10, 2015.  Some of you have seen firsthand these improvements.  Your witness to his new abilities & STRENGTH is what you need to write the FDA about!  For those that have not seen Owen of late, you have witnessed through these updates all the amazing tasks he is doing as he gains strength right before our eyes!

At the end of this message is a template for you to use to write your letter to the FDA or you can choose your own words to write to the FDA. 

But first read our letter – simply this drug is working in ways no one thought possible.

--------------------------------------------------------------------------------------------------------------------------------------------------------------------
~TONY & JEN DUMM LETTER TO THE FDA
Dear Ms. Moon Hee V. Choi
The following is our written testimony submission for the FDA Advisory Committee Meeting to consider Eteplirsen - January 22, 2016.  Our son, John Owen Dumm, (12) started the Sarepta 204 Eteplirsen study on 4.10.15.  He was diagnosed at age four (4) and since then we watched every muscle in his body weaken.  He stopped walking at age eight (8) and early 2015 his arm strength was diminishing quickly.  The following are things he no longer was able to do prior to 4.10.15:
          -He was no longer able to sit up straight
          -He was no longer able to bring his fork to his mouth
          -He was no longer able to scratch his ankle and sit back up again
          -He was no longer able to lift his arm to his face
          -He was no longer able to control his torso
          -He was no longer able to control his legs in any type of position
          -He was no longer able to wiggle his legs
          -He was no longer able to stand with his KAFO’s for more than one hour
          -He was no longer able to reach across the table to grab his napkin
          -He was no longer able to pick up his plate to clear it from the table.
          -He was no longer able to gather his own books at school from his desk
 
Duchenne has robbed our son of daily life functions.  It also dampened his spirits and diminished our hope for at the very least some type of quality of life.  Every day is a battle to move, dress, eat, function and thrive.  Duchenne is a muscle killer, it shows no mercy and stops for no one.
 
Today, 1.2.16, the battle though still with us, the burden has been eased through the weekly infusions he receives of Eteplirsen.  We have experienced ZERO Negative side effects in the 39 weeks he has received this drug.  This drug is SAFE.  There has been absolutely no medical issues or cause for any concern while on this drug.
 
We have, however, witnessed strength in all aspects of his body.  Strength we never thought we would ever see.  When John Owen was diagnosed the doctor said, if a drug becomes available in his life time, it will sustain him at whatever state he was at once receiving the drug.  That doctor was naive to the fact of what Eteplirsen is able to do.
 
On Eteplirsen John Owen has gained on his abilities, not just sustained them.  Since 4.10.15 we have witnessed the following changes in John Owen Dumm:
-Today he can reach down and scratch his ankle and sit back up
-Today he can stand in his KAFO’s for two to three hours without fatigue
-Today he can  reach across the table to steal his dad’s food to play a prank on him
-Today he can  clear his own plate from the table, take it to the counter and set it on the counter
-Today he can  lift a box of Kleenex from his foot rests to his desk
-Today he can  get his books out of his desk while balancing on his yoga ball
-Today he can  keep his legs in an upright position while lying on his back
-Today he can  control his hip flexors so his legs don’t flop open like butterflies
-Today he can  reach up and scratch his face
-Today he can  sit up straight and tall
-Today he can  take his fork to his mouth to eat and not his mouth to his food
-Today he can  peddle his therapy bike on his own for short bursts
-Today he can  reach his hand to his ear and clean out his ear all by himself
-Today he can  draw, color, write without fatigue
-Today he can  he can wiggle his legs as a sign of discomfort while at the dentist
-Today he can  show confidence in using his muscles – he attempts to do things for himself
-Today he can  witness his LVEF % INCREASE instead of Decline
-Today he can  hope for a future that is bright, not cut short at age 19 because his heart is healthy, his lungs are strong and his muscles are GAINING STRENGTH right before his eyes.
 
We urge you and your team to approve the NDA for Eteplirsen as soon as possible.  Everyday Duchenne boys that can benefit from 51 and are not receiving this drug are simply dying.  Eteplirsen is safe, this drug has reversed the course of this muscle killer in John Owen Dumm, and it can and will do the same for every child that can use this drug.

In addition, by approving this drug, you will change the future for not just our boys but for all affected by muscle disease.  Exon skipping is a game changer, this safe and effective drug will change the path of this killer as we know it and will open the doors to many therapies that can and will improve the lives of our citizens.
 
We appreciate your dedication to this process and urge you to approve the NDA for Eteplirsen so John Owen Dumm can continue to build strength.  Now is the Time!
 
Tony & Jen Dumm
5715 Bunker Road
North Royalton, Ohio 44133
John Owen Dumm’s Parents and founders of John Owen’s Adventure, Inc
-------------------------------------------------------------------------------------------------------------------------------------------------------------

You can feel free to write from your heart about why it is so critical for the FDA to allow this New Drug Application (NDA) to pass.  Many more boys can benefit immediately if this drug is approved and your written statement can be the one that derails the path of Duchenne in our boys!

We thank you in advance for your willingness to submit this urgent statement – please send this on to everyone you know, in every form of media (email, social, text, heck pick up the phone and call!)  and ask them, on behalf of John Owen Dumm and John Owen’s Adventure, Inc, to submit their plea. 

IT IS CRITICAL THAT YOU SEND USE THIS EMAIL TO, USE THIS SUBJECT LINE AND THE FIRST LINE AS INDICATED HERE:  Must be submitted by THURSDAY 1.7!!

Send your email to: [email protected]
Email subject line: Written testimony submission for FDA Advisory Committee Meeting to consider Eteplirsen - January 22, 2016
Attn: Moon Hee V. Choi
‘The following is my written testimony submission for the FDA Advisory Committee Meeting to consider Eteplirsen - January 22, 2016.”
 
Here is a sample of what to write OR you can write in your own words (as long as the above is included)
 
~YOUR SAMPLE LETTER
--------------------------------------------------------------------------------------------------------------------------------------
Dear Ms. Moon Hee V. Choe
          The following is my written testimony submission for the FDA Advisory Committee Meeting to consider Eteplirsen - January 22, 2016.  I am writing on behalf of John ‘Owen’ Dumm (12) who has Duchenne Muscular Dystrophy and his foundation, John Owen’s Adventure, Inc.  Owen started the Sarepta Trial 204 on 4.10.15.  He has experienced zero negative side effects from being on this drug.
 
I have memories of Owen before this muscle killer robbed his legs from walking and before this killer took full hold of the rest of his muscles.  I remember him XXXXXXXX  {HERE IS WHERE YOU CAN ADD YOUR EXPERIENCES – for instance, walking in the halls at school, playing in the creek with his brothers, playing the games at his annual fundraiser, JOA Picnic in the Park}.  Duchenne has brutally taken away these abilities in Owen.  I thought that once these muscles were gone, they would be gone forever.
 
This, however, all changed when Owen started this trial.  From all reports he has had ZERO negative side effects.  {IF YOU HAVE PERSONAL EXPERIENCE LIST OUT ALL YOU HAVE SEEN HERE….} I have witnessed the muscles that were weak, get stronger when Owen XXX XXXX    {EXAMPLES – LIFTED HIS FORK TO HIS MOUTH – MOVED HIS BOOKS ON HIS DESK  - SAT UP STRAIGHT IN HIS  WHEEL CHAIR – BENT DOWN AND SCRATCHED HIS LEG….}
 
This drug is working in Owen.  Eteplirsen has proven to be safe in him and in other boys.  I urge you to consider the safety and effectiveness of this drug and insist on the approval of the NDA before one more day goes by and yet we lose one more boy to this muscle killer.
 
Exon Skipping is a game changer for our Duchenne boys.  It is clear from the abilities shown in our Owen that this therapy works.  It will work for all boys that can benefit and approval of this drug will open the doors to cure not just these boys, but all that will benefit from Exon Skipping, in this generation and the next.
 
I fully understand it is your duty to complete this due process.  It is also your duty to supply our citizens with drugs that are safe and effective.  Eteplirsen is both and therefore it is your duty to approve this drug immediately, Now is the Time!
 
I thank you for your consideration.
 
XXXX NAME
XXXX ADDRESS
--------------------------------------------------------------------------------------------------------------------------------------------------------------------
Thank you again so very much for your ACTION!  Please share this with your family, friends, workmates – all you know – we need to get this word to the FDA – This is our country – Our Choice – this Drug WORKS!!! God bless you, Happy New Year and GODSPEED ETEPLIRSEN
 
Tony, Jen, Rutger, Owen and Wade!!! xoxoxoxxo

WITNESS HIS STRENGTH!  NOW IS THE TIME!