The final edition aired on ABC on August 31, 2014, again as a two-hour special, beginning at 9PM ET/PT. It was announced on May 1, 2015 that the MDA would be discontinuing the annual event.
My letter to Dr. Califf this evening
Dear Dr. Califf,
Allow me to introduce myself. My name is Terri Ellsworth, mother of Billy Ellsworth, 15 1/2 years old from Pittsburgh, PA. Billy has Duchenne muscular dystrophy and has been a trial participant for the last five years in Sarepta's 201/202 clinical trial. Billy's first infusion was August 17, 2011. This Wednesday, August 17, 2016 will mark five complete years of weekly Eteplirsen infusions. Billy was in the 30 mg per kg cohort from the beginning of the trial.
It would be negligent of me as his mom to not write you and tell you of his continued success while on the Eteplirsen drug. He continues to do well and remains 90-95% self sufficient and independent as the second oldest boy in the trial. Just today, he was home alone from 9:30-3:30 due to work schedules and lack of outside caregiving. I'd prefer not to leave him alone this long and it doesn't happen often but he has the self-confidence and stabilization that he is okay to be by himself for this length of time. You see, our life is not the typical Duchenne lifestyle. Billy can still dress himself, feed himself, bathe himself, toilet himself, and much more due to his Eteplirsen treatment. I prepared lunch for him today and he had to retrieve it from the refrigerator, heat it in the microwave, and then carry it to his tray table. He then will carry the dishes to the sink. He will also put his beagle on the lead outside during the day for his break and then return him back into the house after removal from the dog lead. This isn't typical Duchenne behavior or natural history, is it? Also, and very importantly, Billy will wake to use the bathroom nightly. He gets himself out of bed (a high bed that he uses a footstool to climb into) and walks out of his room and down the hall and then puts himself back into bed---all without calling out for help. I'm a light sleeper and heard him do this task last night at about 1 am. This isn't typical duchenne behavior either. Daily, I read posts on social media of parents having to turn their sons in bed 5-6 times each night. It's truly amazing that my 15 1/2 yr. old can still do most things independently as he attested to at our adcom in April. Furthermore, nothing has changed since adcom almost 4 months ago. Billy still walks in and out of the hospital weekly for his infusion. We do not own a wheelchair or powerchair but instead have a lightweight convaid folding stroller/chair for long distance walking such as amusement park, zoo, airport etc. He uses an electric scooter to navigate through his large high school consisting of 3 floors.
I don't profess to be a scientist nor anything close to one, but I do know that this is not the natural history of Duchenne and it's not mere coincidence that 10 boys are still walking after 5 years on this drug. What are the odds of this? For some to even suggest that this is placebo effect or that all of these boys are outliers after 5 years is absurd. These types of opinions and statements aren't logical.
Other statements made in our briefing documents claimed that our boys' stabilization is due to "intensive physical therapy and intensive steroid therapy regimen", and not due to Eteplirsen. I can speak to Billy's dosage of just 21 mgs of deflazacort and zero PT for approximately the past 8 years. Furthermore, school released him from all PT in 2014 writing that he's met, maintained, and exceeded all goals.
Billy is not an outlier. He was an extreme toewalker at the beginning of the trial. This is an indication that end of ambulation is near, but instead, Billy's heels are now much closer to the ground and he remains very ambulatory.
I cc'd Dr. Woodcock on this email because she met Billy in April at our adcom. I'm so glad the she witnessed Billy walking into and out of the ballroom where she kindly stayed afterwards to talk with patients and parents. Billy asked Dr. Woodcock to approve his drug and recently Billy found out that you were the commissioner of the FDA and may be involved in helping to make the decision on his drug's approval. I shared with Dr. Woodcock Billy's fears and anxiety including chest pains the night that we returned home after adcom. I assured him that we're doing all we can to convince the FDA that his drug is working. Billy told me that he'd like to meet with you so that you can see how well he is doing and to answer any questions that you may have. What better way to decide the fate of his drug and his well being than to meet with one or more of the patients!
I've included a recent video of Billy just 2 weeks ago of him stepping up and then down a curb and walking up a long ramp into a restaurant. I also have posted on YouTube (Terri Ellsworth) over 200 videos of various activities showing Billy's success on the drug over the past few years.
When I hear statements from the FDA such as, "this is the way we've done it since the 1960's or 70's", I think to myself, but why does it have to still be this way? Where's the progress and innovation? We are in the 2nd decade of the 21st century. The technology we have today didn't exist in the 60's and 70's, so why are we still following the standards from 40 and 50 years ago? How is this progress? If we continue to follow the standards and clinical trial designs from 50 years ago, tens of millions of rare disease patients will be left behind. I've read that you, Dr. Califf, are a proponent of innovation and a champion for pediatrics and support and encourage patient engagement. I don't subscribe to the thought or belief that approving Eteplirsen will be lowering FDA's standards. I don't consider 5 years and over 4000 weekly infusions without any safety issues, 4 biopsies per patient as fast tracking or lowering standards. The boys are producing the missing dystrophin protein that causes Duchenne. Even this small amount produced has had a profound effect on their quality of life as I reported above in regards to Billy. Who should have the right to claim that such a small amount is insignificant, unimpressive, or not meaningful? To a Duchenne patient and family, it is very meaningful and has had a profound effect on quality of life. Shouldn't the Duchenne patient have a voice on how much is meaningful to him? Shouldn't the Duchenne patient have a voice over risk vs benefit.
The science is there and it will get better. We had 13 Duchenne experts speak at adcom who have dedicated decades and their entire careers to Duchenne, including the doctor who discovered the gene in 1986 responsible for Duchenne. They testified that they believe this drug works and that they want to offer it to their patients. In addition, 36 Duchenne experts signed a letter supporting Eteplirsen also stating that they'd never seen a drug provide such benefit in slowing the progression of the disease. Furthermore, Eteplirsen is the perfect example and candidate for FDASIA that Congress enacted 4 years ago.
We have a safe and effective drug now and it would be criminal, immoral, and unethical to allow an entire generation of children die because certain t's weren't crossed or i's dotted. Please don't let this generation of boys be the forgotten boys. I ask you to do the right thing for the boys, girls, and young men and women and to give them a chance at a better quality of life that they all deserve. Wouldn't you want this drug as a life preserver if this were your child or grandchild?
Thank you for taking the time to read this note.
Mediocre people on all levels. Most managers are more interested in covering their #$%$ and play politics than do anything productive. Think that all customers are simply stupid.
The Riceen Letter was sent out early because Riceen was taking a trip to France to have a meeting with some of his followers. He did say he was very proud of his predictions that week and it was 92% right. That was pretty good in his book. He wrote that on Thursday there will be a stock correction with the Dow downd 264 points. But, he also said that Friday will be a buying opportunity. And the market will be up 124 points. The Dow will be down for the week over 100 points.
He writes that SRPT will be higher by Friday after going down on Thursday $1.43.
Monday. Closes at $37.42
Tuesday Closes at $38.43
Wendesday closes at $36.79
Thursday. Closes at $35.33
Friday. Closes at $37.79
Riceen also told his followers that SRPT will be in the 40's by the end of May.
Todd is an epitome of 'incompetence'. His judgment is bordering with dogma - a mediocrity that is so common to MF.
Derepression of DUX4 in skeletal muscle has emerged as a likely cause of pathology in facioscapulohumeral muscular dystrophy (FSHD). Here we report on the use of antisense phosphorodiamidate morpholino oligonucleotides to suppress DUX4 expression and function in FSHD myotubes and xenografts. The most effective was phosphorodiamidate morpholino oligonucleotide FM10, which targets the polyadenylation signal of DUX4. FM10 had no significant cell toxicity, and RNA-seq analyses of FSHD and control myotubes revealed that FM10 down-regulated many transcriptional targets of DUX4, without overt off-target effects. Electroporation of FM10 into FSHD patient muscle xenografts in mice also down-regulated DUX4 and DUX4 targets. These findings demonstrate the potential of antisense phosphorodiamidate morpholino oligonucleotides as an FSHD therapeutic option.Molecular Therapy (2016); doi:10.1038/mt.2016.111.
PMID: 27378237 DOI: 10.1038/mt.2016.111
Purpose, of course. Every MF article has no bottom line - all of them infest the doubt. It's pseudo-knowledgeable business that fools naive investors,
A true test will come shortly when the FDA decides on Sarepta Therapeutics' (NASDAQ:SRPT) DMD drug eteplirsen. The agency was scheduled to make a decision last month, but delayed that decision indefinitely, and subsequently asked Sarepta Therapeutics for additional data.
The original data package was less than perfect, mainly due to a small number of patients in the phase 2 trial that was never really designed as a pivotal trial. The FDA's committee of outside advisors recommended against approving the drug, but the fact that the FDA didn't issue an outright rejection hints at the FDA's willingness to be flexible. Sarepta Therapeutics has already started a phase 3 trial, and has plans for an additional trial that might make the FDA willing to issue an accelerated approval if the new data satisfies regulators.
If the FDA approves eteplirsen, Sarepta's shares will soar. More importantly, it'll give investors confidence in the overall ability of biotech companies to get drugs past regulators.