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Sharon Hesterlee Female Tucson, AZ United States: Favorite

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Duplications

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Sharon Hesterlee's Discussions

Japanese company to start exon-53 skipping trial (morpholino)

Started May 9 0 Replies 0 Favorites

Muscle function recovery in golden retriever muscular dystrophy after AAV1-U7 exon skipping

Started Sep 18, 2012 0 Replies 0 Favorites

French investigators have demonstrated that antisense oligos delivered with a gene therapy approach can improve muscle function and structure over the short term in dogs that lack dystrophin,…Continue

Tags: oligos, gene therapy, exon skipping, dystrophin & utrophin

Connect Meeting Scientific Conference--Informal Poll

Started this discussion. Last reply by Luis Cordero Feb 3, 2011. 6 Replies 0 Favorites

Hi Everyone-- We are in the process of planning for next year's Connect conference (already!) and I would really appreciate some feedback from some of you who attended the conference in Denver this…Continue

Research presentations at the Connect Conference

Started this discussion. Last reply by Ofelia Marin Jun 30, 2010. 7 Replies 0 Favorites

We are just putting the final touches on the agenda for both the Connect Conference and the first annual DMD Therapeutic Development conference. Both conferences will take place in parallel in…Continue

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Sharon Hesterlee, PhD, Sr Director of Research

Profile Information

About me:: I joined PPMD at the start of 2010 after 11 years developing and overseeing the translational research program at the Muscular Dystrophy Association. I'm thrilled to be able to focus all of my time just on DMD/BMD and can promise you that I'm always thinking about your sons.

About my family:: My very patient husband Scott is the Curator at the Pima Air and Space Museum in Tucson and is frequently "Mr. Mom" when I'm out of town. Our son Pierce is five--he has been diagnosed with an autism spectrum disorder.

City:: Tucson, AZ

Latest Activity

StaffSharon Hesterlee posted a discussion

Japanese company to start exon-53 skipping trial (morpholino)

A Japanese company has announced that it is starting a clinical trial for an exon 53 skipping morpholino. I don't have any more details beyond this notice, but would think it's highly unlikely that any out-of-country participants would be considered just based on how the Japanese regulatory system works.(both Sarepta and Prosensa are also in the preclinical stages for an exon 53 skipping drug)Press release: …See More

May 9

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StaffSharon Hesterlee commented on PPMD's blog post Report from MDA's Scientific Conference 2013

"Ofelia--he didn't go into that much detail (as far as correlating nNOS binding restoration with particular dystrophin outcomes). It was almost entirely the same information that Sarepta has already presented--I don't think he was…"

Apr 25

StaffSharon Hesterlee commented on PPMD's blog post Report from MDA's Scientific Conference 2013

"So, actually your question was about treatment over time and I don't have detailed enough notes to answer that question, but certainly the stunted growth you see in steroid-treated mice (and not in VBP15-treated mice) would suggest that your…"

Apr 25

StaffSharon Hesterlee commented on PPMD's blog post Report from MDA's Scientific Conference 2013

"Hi Amit--so basically the drug didn't show some of the specific negative effects you see with prednisone (prednisolone actually) in the mdx mouse. Steroids don't work as well in the mdx mouse as they do in humans although it's…"

Apr 25

StaffSharon Hesterlee replied to Bernardo A. Iriberri's discussion Ataluren Webinar

"Berit, we hope that this document will help speed the approval of all therapies. We will coordinate with our colleagues in Europe on an EMA effort (although in some key ways the EMA is more progressive than the FDA already)....all of these…"

Apr 23

amit gupta commented on Sharon Hesterlee's blog post Drug Development Prospects for Duchenne: The Pipeline is rich and deep, with lots of forward momentum

"OK. I had corresponded with Dr Ruegg earlier and he said the PPMD funding was for preclinical mdx work. I am hoping he would present data at the convention to proceed further."

Apr 18

StaffSharon Hesterlee commented on Sharon Hesterlee's blog post Drug Development Prospects for Duchenne: The Pipeline is rich and deep, with lots of forward momentum

"Sorry--that list isn't comprehensive. There are probably twice that many additional things, particularly in the preclinical space, that could be added and I have a more comprehensive version that I work from internally. We just…"

Apr 18

amit gupta commented on Sharon Hesterlee's blog post Drug Development Prospects for Duchenne: The Pipeline is rich and deep, with lots of forward momentum

"Thanks Sharon. I didn't see its mention in your blog, hence the q."

Apr 18

StaffSharon Hesterlee commented on Sharon Hesterlee's blog post Drug Development Prospects for Duchenne: The Pipeline is rich and deep, with lots of forward momentum

"Hi Amit: Pat Furlong and Bob McDonald (Board Chair) and I will all be at the MDA conference next week. PPMD is already funding the tamoxifen project ($100K), along with UPPMD. Sharon "

Apr 18

amit gupta commented on Sharon Hesterlee's blog post Drug Development Prospects for Duchenne: The Pipeline is rich and deep, with lots of forward momentum

"Thanks Sharon. Do you have any thoughts about funding Tamoxifen? I believe Dr Ruegg will be at the MDA convention next week looking for funding for clinical trial. Is PPMD attending?"

Apr 18

StaffSharon Hesterlee posted a blog post

Drug Development Prospects for Duchenne: The Pipeline is rich and deep, with lots of forward momentum

This spring brings news of progress to the Duchenne community as Sarepta engages in talks with the Food and Drug Association (FDA) to determine if they agency will review an application for accelerated approval for the exon 51 skipping drug eteplirsen and releases its 74 month data showing…See More

Apr 9

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StaffSharon Hesterlee replied to Bernardo A. Iriberri's discussion Ataluren Webinar

"We can try...and will try. If we have to do a European survey, though, we will do it (partnering with colleagues in other organizations in Europe). One way or another it will get done. "

Mar 18

StaffSharon Hesterlee replied to Bernardo A. Iriberri's discussion Ataluren Webinar

"Hi Berit--My colleague Holly Peay is doing the majority of the work on the risk/benefit survey (along with all of the parents who have been willing to complete this hour and a half long survey!). We are analyzing the preliminary results now,…"

Mar 14

StaffSharon Hesterlee replied to Bernardo A. Iriberri's discussion Ataluren Webinar

"Hi Peter, I think the issue is just the same as in the US--even if the EMA granted access through some kind of compassionate use program, PTC would be prohibited from selling the drug and would have to provide it for free (and monitor for adverse…"

Mar 1

StaffSharon Hesterlee replied to Bernardo A. Iriberri's discussion Ataluren Webinar

"Hi Berit-- We are in the process of finalizing a white paper now that pushes the FDA on issues of flexibility in trial design and faster approvals for rare disease--it compares all the new proactive legislation with what they are actually…"

Feb 28

StaffSharon Hesterlee replied to Bernardo A. Iriberri's discussion Ataluren Webinar

" Hi Berit--I don't know the time line but am happy to ask PTC Therapeutics if they know it. In theory we might be able to persuade the EMA. Pat and I just spent a week in Europe meeting with various European funding groups…"

Feb 26

Sharon Hesterlee's Blog

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Drug Development Prospects for Duchenne: The Pipeline is rich and deep, with lots of forward momentum

Posted on April 9, 2013 at 9:00pm 5 Comments 0 Favorites

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A Strong Start to 2013

Posted on February 4, 2013 at 2:30pm 0 Comments 0 Favorites

Thanks to everyone who contributed so generously to PPMD’s holiday appeal we have been able to fund three new projects designed to speed the progress of the clinical trials underway in Duchenne. As many…

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Sarepta Summary of Outstanding Questions from PPMD Webinar

Posted on January 17, 2013 at 12:00pm 0 Comments 0 Favorites

On October 23, 2012, PPMD hosted a webinar with Sarepta to discuss of the 48 Week dystrophin and 6-minute walk test (6MWT) data from the Phase IIb extension study in Duchenne as well as next steps for the eteplirsen program. Chris Garabedian, President and CEO of Sarepta…

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Duchenne Drug Development Round Table

Posted on November 15, 2012 at 10:00am 5 Comments 0 Favorites

There are a lot of frustrations in the Duchenne community—one of the most common themes I’ve heard is “why isn’t all this research and drug development coordinated better?” The problem, of course, is that there are a lot of companies and funding groups trying to help at once—sometimes it may feel like too many…

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At 6:55pm on April 12, 2012, kimmy watters said…: hi sharon i want would like to say thank you very much for working so hard to help our boys that one day we will find a cure . i have been seeing lots of comments about the drug avi how is going to help our boys and how soon will get it fda approve. thanks kimmy

At 7:22pm on November 29, 2011, kimmy watters said…: thank you sharon that explain alot i would to be able to watch the web on wed but i am on dial up very slow but am great ful it still work. i am also very exctied that about the ophan drug. will that help alot of our boys. will it help them live longer.kimmy

At 6:44pm on November 28, 2011, kimmy watters said…: hi sharon my name is kimmy watters andi have a question, the sweet young boy brady 14 lives ca. what is the fat emoby sorry i do not spell well. he was such a sweet kid very handsome as well thank you kimmy watters

At 11:54am on October 6, 2011, Suphareck Ratiromphan said…: Moreover, are there any medicine or treament for slowing the progression of Duchenne for my brother??...

At 11:50am on October 6, 2011, Suphareck Ratiromphan said…: Dear...Sharon,

Thank you so much for all your supports and suggestions. It helps me and my family so much, especially about the heart specialist for my brother. I'm very appreciated for your kindness. If there's a chance that the community coming abroad to Thailand, that would be very great as well because there're actually alot of people who face difficulty with the Duchenne. Moreover, once again thank you so much. And, if there're any more problems or questions, would you mind to ask u again ??.

Sincerely yours,

At 8:22am on October 5, 2011, Suphareck Ratiromphan said…: Dear...Sharon,
My brother, he cannot walk anymore, cannot move any parts of his body except he can still shew some food, talk as normal people, and move his head. But he can't eat alot as others. Also, he cough alots. He needs to use the respirator all the time otherwise he's hard to breath by hisown. Unfortunately, there are not any treatments in Thailand. Therefore, he hasn't taken any medicine or done anything about medication. Several times, he can't breath so my parents take him to the hospital for getting some supplimentaly foods. Accordingly, What should i do??I really want him to do some treatments or make him be better.. Thank you for your kindness

At 8:22am on October 5, 2011, Suphareck Ratiromphan said…: Dear...Sharon,
My brother, he cannot walk anymore, cannot move any parts of his body except he can still shew some food, talk as normal people, and move his head. But he can't eat alot as others. Also, he cough alots. He needs to use the respirator all the time otherwise he's hard to breath by hisown. Unfortunately, there are not any treatments in Thailand. Therefore, he hasn't taken any medicine or done anything about medication. Several times, he can't breath so my parents take him to the hospital for getting some supplimentaly foods. Accordingly, What should i do??I really want him to do some treatments or make him be better.. Thank you for your kindness

At 8:35am on February 27, 2011, pop jarvis said…: If you are interested, please join in the discussion about electrostim treatment at South Texas Innovative Medicine Discussion Forum. My daughter has RSD and went to Dr Rhodes and STIM. The "treatment" didn't work at all for her. The STS has been on the market for almost a DECADE! I'm suspicious that there's never "time" to assess the success rate data...

At 3:01pm on January 14, 2011, Jason Darienzo said…: Hi Sharon, NIH article http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2910579/ is about

preventing calcium leak in DMD significantly reduced muscle damage.

A company named Armgo Pharma inc. in New York State has 2 drugs in phase II trials in Europe for the heart muscle and 1 drug of muscles disorders in development.

Do you have any more info about this?

At 2:13am on September 21, 2010, Char Burke said…: Hey Sharon, Just wondering if you could give feedback on Acceleron and their recent infusion of $$ from Shire @ 480mil. Our son has a duplication of 54-57 and exon skipping is not an option b/c of the duplication. I was watching Biomarin but they dropped out. Now Acceleron. Biomarin was trying to turn on utrophin vs. Acceleron is trying to work with myostatin. Any other things I should be watching for duplications? Thanks Char Burke - mom to Will age 8.