Top Tweets for #EndDMD
Hey @US_FDA #RealWorldEvidence is Evidence.
When rare disease patients SHOW you that a drug works, Believe them.
@FDA_KyleD @FDACommissioner
#EndDMD #EndDuchenne #Duchenne
Billy yesterday, at 25 1/2 years old with #DMD, still thriving and benefitting from #eteplirsen after 15 years on treatment. Impressive PUL and ROM at this age. Defies natural history. @US_FDA
13 yo with #Duchenne is swimming!!! Real World Evidence is Evidence.
Bring back common sense to regulatory policy.
@FDA_KyleD @FDACommissioner @US_FDA @FDACDERDirector
@POTUS @SecKennedy
@SenRonJohnson @SenGillibrand
#rarediseases #EndDMD
Posting everyday until
@FDA_KyleD @FDACommissioner @FDACDERDirector signals @PTCBio to say #realworldevidence will be used for #ataluren approval This is what 9 years on Ataluren looks like, this is not the natural history for a 13 yr old with #duchenne #timeismuscle @SecKennedy
โMom, you have to do this for me,โ he told her. โYou have to do this for other little boys like me. And thank you, Mom, for giving me a fighting chance.โ
My son is my biggest encourager in my advocacy work. It's God, Ryu, and my two brothers I've lost to Duchenne that keep me going in this work day after day. I'm so grateful toย @Samantha_Kamman for beautifully writing my families story for theย @ChristianPost.ย https://t.co/E0UVpG46NP
@andrewpannu $SRPT Here's some research; In late May '23 SPRT DMD GT will likely be approved for 1/2 the US population (ambulatory). 4-6Qs after; EU and then non-ambulatory follow. Lets say 3M/dose and 30k pts, so $90B TAM. PFE DMD GT is not safe competition.
#JerrysKids #EndDMD
Next Sunday Isaac & #TeamArchie will be pounding the streets @dublinmarathon . They are completing their 4th Marathon in Memory of Archie. Isaac wants to honour his MAGNIFICENT Big Brother. Mile 16 is for Archie. Wear something orange if you can #ArchiesLegacy #Forever16 #EndDMD
Tonight is Bubba's BIG Day. Graduating with the Class of '22 from @creekwoodhs.
#CureDMD #ENDDMD I am a #DMDDAD
#GRADUATIONDAY #ProudDad
As a family we are all still massively struggling with the very sudden loss of Sadie in November Today, George, Isaac and myself were talking of her, as we often do:
"Mummy, I miss Sadie as much as I miss walking and playing football."
When will Governments step up? #EndDMD
It's Day 7 of Duchenneber โญ๏ธ
"I'm looking forward to my visit from Santa at Christmas. I can't wait to leave out a mince pie & a carrot for Rudolf!" Elliot age 4
Give boys like Elliot the gift of hope this year by donating to vital DMD research: https://t.co/r2cB5XGpb4
#enddmd
$SRPT Iโve been in this stock longer than I recall. I was in AVI in the early days. I was here before @douglas_ingram or @ianestepan. I was before DMD and CG. I Have been a big supporter for the fight to #endDMD.
And then the bell rings. He is off for the day. Then the day passes without a call or urgent email and he exits the building with rosy cheeks, smiling eyes and a joy that has been missing since the pandemic began. โI think I made a friendโ. Today is done. #EndDMD
#desafioduchenne @dppspain #rarediseases #torreglories #dmd #wdad2020 #duchenneawareness #duchenne #enddmd en Barcelona, Spain https://t.co/srpLQtYG6o
$SRPT it is with great pleasure that we announce our goal has been meet. THANK YOU to all that helped reach the goal and support a great cause to #endDMD
https://t.co/rPnie84EyH
$SRPT tomorrow is the BIG day... Iโm getting married. In lieu of wedding gift we are asking for donations. Here is the great part, even thou I didnโt invite #biotwitter you can still donate and help to #endDMD ๐๐
https://t.co/rPnie8mfXh
A year in pictures.
May 2020 be as inspiring and creative, with a touch of mystery and a ton of hope.
#MyArtistBoy
#EndDMD
$SRPT today at CS fireside chat Doug said, wrt manufacturing, Brammer build-out was complete, Paragon has even more capacity and SRPT has their own in-house manu also. Company SPOX confirmed "this is to support dosing thousands of patients a year" #endDMD
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