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Sharon Hesterlee
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- Tucson, AZ
- United States
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Sharon Hesterlee's Discussions
Japanese company to start exon-53 skipping trial (morpholino)
Started this discussion. Last reply by Sharon Hesterlee Sep 8, 2014. 2 Replies 0 Favorites
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…Continue
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
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
Sharon Hesterlee's Blog
Without Endings There Are No Beginnings…
Posted on January 5, 2015 at 1:30pm 1 Comment 0 Favorites
It’s New Year’s Day 2015 and I think Janus, the two-headed Roman God who looks forward and backwards at the same time, is a fitting representation of my life right now. I need to tell you all that I have had to make a difficult decision this year—at the end of this month I will be winding up my time at PPMD while simultaneously starting mid-month with the Myotonic Dystrophy…
Now is the time for strength.
Posted on December 18, 2014 at 3:02pm 0 Comments 0 Favorites
We’ve been working toward this moment for years. And the time for action is now.
When I started with PPMD in 2009, Pat Furlong and I talked a lot about what it would take to end Duchenne. We knew it wouldn’t be one thing, and we knew it wouldn’t be easy. We believed that only a combination of therapies could address the entire disease within the body.…
No Exon Left Behind
Posted on July 21, 2014 at 10:15am 6 Comments 0 Favorites
I'm often asked by parents to explain exon skipping. In fact, at last month's 20th Annual Connect Conference, after we announced a grant awarded to Dr. Judith van Deutekom, parents wanted to know what it is we are actually funding.…
ContinueNew Duchenne Therapies in Development
Posted on April 24, 2014 at 12:30pm 3 Comments 4 Favorites
As we finalize the agenda for this year’s Connect Conference, I am once again amazed at how much of a challenge it is to squeeze in all of the talks on different therapeutic approaches to treating Duchenne—in fact, there are too many to cover comprehensively this year. Sometimes it’s good to remember that mutation-specific approaches like exon skipping, although promising and…
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Hi Sharon, Thanks for all the help that PPMD has been offering. My son (4.5 years old) has regular duplication of exon 51-55. Can you please help me find any clinical trials that he can participate in. Thanks, Satish
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
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
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
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,
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
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
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...
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?
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