Dr. Anne Connolloy's Testimony
I’m a neurologist at Washington University in St Louis and I’ve worked with children and adults with neuromuscular disorders for 27 years. I have been a consultant for Sarepta, but stand to gain nothing financially from approval. Please consider what I have to say from the perspective of a clinical researcher in Duchenne dystrophy, a neuromuscular pathologist, and finally with their permission I speak for Justin and Cole who I’ve followed for three and a half years in the extension study.
I know well the difference between Duchenne and Becker muscular dystrophy as I have cared for more than a hundred and fifty boys and men with these disorders. If I have a question whether a boy has Duchenne or Becker I do in fact assess the number of dystrophin positive fibers. In the recent FDA briefing it was stated that the percent positive fibers is not a reliable way to quantify dystrophin. Not only do I disagree with you I ask you to review those biopsies carefully and note that the fibers with dystrophin are larger and more frequent than any biopsy I’ve ever seen with revertant fibers. I believe these dystrophin fibers are driving the clinical effect. Furthermore, because Justin and Cole are so much stronger than I would have expected, if I met them for the first time today I would have suggested they have muscle biopsies. When I consider the post Eteplirsen biopsies and their physical examinations I would have to classify them as having Becker muscular dystrophy. Thus, I do believe that dystrophin positive fibers are a clear biomarker for strength and rescue of muscle.
Now a minute on behalf of Justin and Cole. I have followed Justin since the age of three and treated with intermittent , twice weekly steroids. However, at the age of 11 when he entered the study he had difficulty getting off the ground and I timed him at 26 seconds and he subsequently lost the ability to get off the ground. Based on all natural history that you and I have reviewed he should have stopped walking by age thirteen. At age 16 and a half, after recovery from a femur fracture, he is still walking.
My second patient, Cole, was 10 years old when he started the trial and has also done well despite a fracture at age 11 and a half requiring a cast and no weight bearing for eight weeks. He has regained the ability to walk and continues to do so at 14 and three quarters years. They are both exceptionally bright.
These two teenage boys do not require someone to feed them, take notes, or take them to the bathroom. While I am a strong advocate of corticosteroids, make no mistake about this fact, corticosteroids whatever the regimen do not explain the data here. Be careful of type 2 error.
She just made finished at exactly 2 minute and 59 seconds when Caleb “The Axe” Alexander cut off the mic.
This is all you need to know about whether Eteplirsen works or not. Alexander, whose eyes were glazed over at this point, was probably busy playing games on his lap top during the testimony.
mjrruby2000000 • May 3, 2016 3:49 PM Flag
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Been around for 20 plus years ...
I've never seen shorts this frightened. This drug will be approved in some fashion. Guaranteed. It matters not what these shorts have to say.
If they believe that upper management are ready to commit political suiside, they're nuts. Approval is for sure. No question.
Sentiment: Strong Buy
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onewaytomars2 • May 3, 2016 3:53 PM
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How many times have you been wrong before (in case of SRPT only)?
Brilliant mind. Thanks. Shorts must read your posts. If they are smart.
By ignoring 'careful_investor_wins' I've cleaned a bunch of pages and my headache is gone.
Imagine: if only one boy (happened to be the son of one of the FDA boss's) - had a duchenne, Sarepta's chart would've been similar to a smoky tail of the rocket.
'Recent FDA appropriations bills passed by the House and Senate appropriations committees included report language reminding the agency it has “the tools, authorities, and latitude necessary,” to approve rare disease treatments like drugs for Duchenne as fast as possible.'