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Please Tell the FDA to Stop Postponing Their Decision re: Eteplirsen- Children Will Continue to Die in the Meantime

Health & Wellness  (tags: DMD, Duchenne, death, children, FDA, cure, research, government, petition )

- 2064 days ago -
Please take a second to send this email. DMD is fatal, but a new (& safe!) drug could be a cure! The FDA have reversed their previous promise based on a completely different drug. Children will continue to die if this decision continues to be postponed


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Lauren S (26)
Tuesday November 26, 2013, 6:11 am
If you skip down to 'PLEASE HELP' on the link it'll tell you how to send the email, but I'll post it here as well.
Here is the full body of the text to be copied and pasted into the email:

Dear Drs. Woodcock, Jenkins, Temple, Unger, Bastings and Farkas,

I understand that the FDA has reversed its previous stance on whether to consider Sareptaís NDA for Eteplirsen. The Agency has cited the recent failure of other dystrophin-producing drugs as one reason to require that Sarepta conduct a large, placebo-controlled phase 3 study before applying for approval. I do not understand this reasoning, as we donít even know how much, if any, dystrophin was produced by the drugs that failed to meet their primary clinical trial endpoints. In contrast, we know that Eterplisen produced dystrophin in all 12 treated patients at levels widely believed to lead to functional benefit. Whatís more, the treated patients have stabilized since the dystrophin took effect. One third of the children actually improved, a clinical outcome that is unheard of in Duchenne.

I do not have a rare fatal disease, but if I had one I would want the freedom to choose a drug in consultation with my physician that has a highly favorable safety profile and early indications of efficacy. I believe the FDAís #1 job is to protect people. While we understand there are certain risks with approving a medication studied in only 12 patients, the risk is a minimal and tolerable one compared to the certain pediatric deaths and losses of ambulation that will occur if you require several more years of study before approving this drug.

I implore you, work with Sarepta to accept the creative and aggressive confirmatory trial design they have planned. Understand that boys will die and boys will lose their ability to walk if this decision continues to be postponed.



Frans Badenhorst (582)
Tuesday November 26, 2013, 6:12 am
problem with the link Lauren......

Error 403

We're sorry, but we could not fulfill your request for /blog/need-your-help-please-in-honor-of-charleys-birthday on this server.

Lauren S (26)
Tuesday November 26, 2013, 6:12 am
And here is the full list of recipient email addresses:,,,,,,,

Lauren S (26)
Tuesday November 26, 2013, 6:13 am
Oh gosh! Sorry Frans, I don't know what's happened- it's working on mine :/
I'll see what I can do. I might try resubmitting again if it'll let me :)

Lauren S (26)
Tuesday November 26, 2013, 6:17 am
In the meantime, here's the link:

Animae C (508)
Tuesday November 26, 2013, 6:22 am
Separate emails sent to;

Will also forward ASAP

Thank You Lauren

Animae C (508)
Tuesday November 26, 2013, 6:35 am
The link works fine for me.

Lauren S (26)
Tuesday November 26, 2013, 6:38 am
Hi Frans,
I've just tried the link on my both my brothers' laptops and it's working on theirs as well- would you mind telling me if the link still isn't working (if you have the time!)?
Is the link not working for everyone else as well? :/

Lauren S (26)
Tuesday November 26, 2013, 6:40 am
Oh, thanks Ani Mae- didn't see that before I posted! :)

Past Member (0)
Tuesday November 26, 2013, 7:18 am
Emails sent-thx Lauren

Annemie O (153)
Tuesday November 26, 2013, 2:47 pm
Noted and done

Past Member (0)
Tuesday November 26, 2013, 2:49 pm

Angelika R (143)
Tuesday November 26, 2013, 2:49 pm
Am about to send out my emails now-just subject line still missing-that's why I need to get from you the CORRECT NAME AND SPELLING of that Et...drug as it's been spelled in all the articles there in various ways-thank you

Angelika R (143)
Tuesday November 26, 2013, 2:55 pm
Never mind-checked www -all emails sent with receit request - had no issues here. Thx Lauren

Past Member (0)
Tuesday November 26, 2013, 2:56 pm
e-mails sent,noted--ty Lauren

Bridget Robertson (106)
Tuesday November 26, 2013, 3:00 pm
This will take awhile. Noted and begun to send.

Angelika R (143)
Tuesday November 26, 2013, 3:02 pm
Side note: sadly, we all know the FDA is also bought/owned by BigPharma and you may also remember the scandals around GSK over vaccines-a quite dubious company.

Birgit W (160)
Tuesday November 26, 2013, 3:57 pm

Athena F (131)
Tuesday November 26, 2013, 5:14 pm
thank you

Vivian B (169)
Tuesday November 26, 2013, 5:28 pm
My stupid mobile won't let me send this. It is because I'm on a mobile. Not the link's fault. But have shared and shared multiple ways.
Wish I could do more. I had a family member with an incurable tumor that died at a young age. I know what it feels like to feel helpless.
Thanks for bringing this to my attention! Hoping for the best!

. (3)
Tuesday November 26, 2013, 6:09 pm
Noted and emails sent. Hope it helps.

Mary T (356)
Tuesday November 26, 2013, 7:16 pm
Sent email - thank you Lauren & AniMaeChi for forwarding

Genoveva M (328)
Tuesday November 26, 2013, 7:29 pm
Email sent.
Thanks Lauren & AniMaeChi for forwarding.

Twyla Sparks (208)
Tuesday November 26, 2013, 7:58 pm

Constance F (418)
Tuesday November 26, 2013, 9:27 pm
yes~ email sent to addresses posted. thank you Sharon and AMC for sending.

Winn Adams (179)
Tuesday November 26, 2013, 10:33 pm
Posted on Facebook

Terrie Williams (798)
Tuesday November 26, 2013, 10:47 pm
Emails sent, noted.

Ljiljana Milic (115)
Tuesday November 26, 2013, 11:01 pm
Emails sent to posted addresses & Thank you Lauren for posting and AniMaeChi for forwarding

Lynn Pascente (141)
Tuesday November 26, 2013, 11:01 pm
Letter sent to all emails individually. Thanks Lauren & AniMae for the forward!

Krish Away (43)
Wednesday November 27, 2013, 12:51 am
Email sent. Hope they 'll see our mails & do something about it.. At-least they'll know someone is concerned about their decisions. Won't they?

Thx Lauren for posting & Ani for forwarding

Krish Away (43)
Wednesday November 27, 2013, 12:53 am
Oh.. & cc'ed

as well.. its available on Original Post

Krish Away (43)
Wednesday November 27, 2013, 12:55 am
By post I mean this..

Past Member (0)
Wednesday November 27, 2013, 1:13 am
Mails sent :)

Kathleen R (192)
Wednesday November 27, 2013, 2:14 am
Sent e-mails and noted. Thank you Lauren and AniMae

Victoria Oakey (124)
Wednesday November 27, 2013, 2:55 am
Sent the emails as requested. Thank you.

Lindsay K (6)
Wednesday November 27, 2013, 4:06 am
Thanks for sharing

Charmaine Shannon (38)
Wednesday November 27, 2013, 4:53 am
Noted and Shared !! Working on this......Thank-You Lauren and AniMae for the FWD !!

John Holton (344)
Wednesday November 27, 2013, 5:33 am
S and N

Angelika R (143)
Wednesday November 27, 2013, 9:01 am

Your message

To: Commissioner FDA
Subject: Duchenne, Eteplirsen and the FDA
Sent: Tuesday, November 26, 2013 5:54:14 PM (UTC-05:00) Eastern Time (US & Canada)

was read on Wednesday, November 27, 2013 11:11:41 AM (UTC-05:00) Eastern Time (US & Canada).


Ruth M (235)
Wednesday November 27, 2013, 9:01 am
Ok will do this when I get home can't do it on my mobile. Thanks AniMae and all others for sharing this

Krish Away (43)
Wednesday November 27, 2013, 10:44 am
Thx Angelika.. your comment was re-assuring to everyone that our mails will be READ
Thanks for posting :)

Barb SiteIssues V (202)
Wednesday November 27, 2013, 11:39 am
Noted & Email sent

Angelika R (143)
Wednesday November 27, 2013, 11:59 am
Krish-I guess that was ONLY because i requested a receit-as I stated above.(perhaps also my subject line MIGHT have mattered..)( I hope not everyone who emails this or another letter were using the same subject line!)

Neill Abayon (667)
Wednesday November 27, 2013, 2:29 pm
Email sent.

Past Member (0)
Wednesday November 27, 2013, 2:32 pm
I have signed the petition.

sylvie C (46)
Wednesday November 27, 2013, 2:47 pm

Angelika R (143)
Wednesday November 27, 2013, 4:00 pm
Your message

To: Farkas, Ronald
Subject: Duchenne, Eteplirsen and the FDA
Sent: Tuesday, November 26, 2013 5:54:14 PM (UTC-05:00) Eastern Time (US & Canada)

was read on Wednesday, November 27, 2013 5:51:32 PM (UTC-05:00) Eastern Time (US & Canada).



Sherri Martin (78)
Wednesday November 27, 2013, 4:39 pm
Noted and Sent Emails.....

Ondine J (134)
Wednesday November 27, 2013, 9:11 pm
Noted and sent emails. Thanks Lauren and AniMaeChi.

cynthia l (207)
Wednesday November 27, 2013, 9:58 pm
signed thx AniMaeChi for forwarding

Sharon Wardle (103)
Wednesday November 27, 2013, 11:07 pm
All done. Truly hope it helps ~ Good luck!

Krish Away (43)
Thursday November 28, 2013, 1:03 am
Good to know Angelika that your mails are read. I got delay notification for ALL except..

health care policy advisor to US Rep. Joe Crowley co-chair of the Congressional Caucus on Rare Disease.

I used a completely different subject line though..
Please Consider Sarepta's NDA for Eteplirsen


kate K (481)
Thursday November 28, 2013, 2:35 am
noted and e-mail sent

Shelli S (374)
Thursday November 28, 2013, 6:01 pm
Sending now-Thanks 4 sending Sharon

Michael A (28)
Monday December 2, 2013, 12:52 am

Krish Away (43)
Tuesday December 10, 2013, 12:44 pm
Email sent with regard to

Hoping For Best..

Charmaine Shannon (38)
Thursday December 12, 2013, 10:14 pm
Have followed up with the E-Mail for the LOSS of another Child and have RE-SHARED this with "ALL" that I know and could think of !!!! This is SO SAD and to think that there are Officials that can "HELP" and are standing in the way......Have You NO Conscious or HEART???? WHAT IF IT WAS YOUR CHILD ????
Prayers that this will be SOLVED for these PRECIOUS CHILDREN and also Prayers for the Children and their Families !!!! Thank-You....

Birgit W (160)
Friday December 13, 2013, 5:46 am
Thanks Lauren.

Veselin Varbanov (23)
Tuesday December 31, 2013, 3:55 am
Noted ,thanks

Nancy C (806)
Saturday January 18, 2014, 12:55 pm
late, but sent...ty!
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