$SRPT I remember the eteplirsen adcom panel and subsequent next day articles on rejection. Every headline stated 'eteplirsen' rejected or failed. The vote was 7-6 against and never read in a headline that it was a close vote. Yesterday's 8-6 vote was 'close'. Go figure.

Infusion number 200 and unstoppable! Not age (14.5), a broken femur + surgery, fractured humerus, bi-lateral tendon release or compression fractures. 🙌 $Srpt #exondys51 #eteplirsen #ClinicalTrialChild #MiracleMed #Duchenne #DMD

$srpt Just a thought... would anybody be shocked if the clinical hold is already lifted (in the past day or so) but DI is holding off on announcing it til Friday so he can bundle the 'good' news with what seems to be a failed EMA appeal for eteplirsen

Does it work? You tell me:
May 2015: Started Eteplirsen
June 2017: Bilateral tenotomy (age 13)
Aug 2018: L Femoral Neck Fracture repaired w ORIF (Age 14)
Oct 2019: R Tibia/Fibula head fracture (Age 15)
Is he still walking? Yes, he most certainly is. $SRPT Sarepta Therapeutics #Exondys51

Power in numbers. I hope this group focuses on awareness for DCVax much like the DMD foundations focused on Eteplirsen.
$NWBO #GBM

Vinay Prasad barttels Michael Joyner
With a nod to @dphdk an *ahem* interesting U.S. FDA label of eteplirsen (Exondys 51).
Marketing authorization granted while explicitly stating lack of evidence:'A clinical benefit of EXONDYS 51 has not been established'
What the fresh hell?

Round number! 1100 citations to this article...

doi.org/10.1016/S0140-…

#eteplirsen #DMD #Duchenne #exonskipping #PMO #clinicaltrial

$SRPT gentle reminder that withdrawal of eteplirsen AA is entirely possible, and from an evidentiary standpoint, more likely than not:

With aducanumab's FDA PDUFA date approaching, it seems like one relevant question is what acting commissioner Janet Woodcock learned from the eteplirsen (Serepta's DMD drug) imbroglio
Thread: 1/n

Pharmdca $SRPT PPMO POC data for 20mpk/mo of SRP-5051 (exon skipping for exon51 restoration of reading frame), as improved mechanism of delivery for Eteplirsen, by the end of the year. People don’t understand the significance and implications (very significant platform relevance), IMO.

For the last decade, #Duchenne 's drug development has been a wild Wild West.

Add to this the gung ho #CRISPR types + u get fatalities. Case involved 3% expression, infinitely more than AA-approved #eteplirsen .

Excuse my political incorrectness. $SRPT

medrxiv.org/content/10.110…

Giving a couple of makeup speeches via zoom to his college professor,including an impromptu speech on an impromptu topic. Next up is a persuasive speech and he chose how he helped to convince the FDA to approve his drug treatment by testifying on behalf of #eteplirsen #DMD $SRPT

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$SRPT
1 example from the time machine...Everyone wins except the long-time FDA staff members who still complain, calling eteplirsen an “elegant placebo”. Perhaps it is time for them to move on and for patients to stop dying in slow and very non-elegant clinical trials.

$SRPT Science Daily: 3 Studies Proves Eteplirsen Slows Down Respiratory Decline. sciencedaily.com/releases/2019/…

Last infusion for 2022 coming up later today, year 12 to start next week. ❤️ #Duchenne #Warrior 🙌🏼👊🏼 #eteplirsen #pioneer #exondys51

$SRPT #Eteplirsen spent a very long time in approval phase and is by no means the longest in AA without confirming. Some over 5 years from AA and counting. See page 5 of 14 for FACTS. I don't see any pounding of chest on the others.
fda.gov/media/88907/do…

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From one of the posters. Based on the most current and comprehensive
follow-up data with adjusted comparisons to
SOC treatment, eteplirsen was associated with
significant and clinically meaningful delays in time
to loss of ambulation. $SRPT

Last infusion for 2022.
592 infusions and 11+ years are in the book without a single adverse event! #duchenne #musculardystrophy #eteplirsen #pioneer #exondys51 #trailblazer #clinicaltrials $SRPT

Exondys51 works its magic again. 14 yo w/ prior bilateral tenotomy in June 2017. Left Femoral Neck Fracture repaired with ORIF Aug. 22. Released to bear weight Sept. 21. AGAIN walking independently. 🙌 youtu.be/uamjTe9Vni4 #dmd #duchenne $srpt #eteplirsen #exondys51

First infusion of eteplirsen to treat #MuscularDystrophy ! It has been a battle to get to this point down to braving the snow, hail and wind. About time things should be looking up. MDA News MDA Philadelphia

Proud to be included in a new brain tumor advocacy collaboration!