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Sharon Hesterlee
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  • Tucson, AZ
  • United States
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Sharon Hesterlee's Groups

Sharon Hesterlee's Discussions

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

Started May 9, 2013 0 Replies

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

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

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

 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

Latest Activity


Staff
Sharon Hesterlee commented on Sharon Hesterlee's blog post PPMD Awards $100,000 in Exploratory Grants
"Hi Amit--I believe they are within a year of starting a trial.  Sharon"
Mar 25
amit gupta commented on Sharon Hesterlee's blog post PPMD Awards $100,000 in Exploratory Grants
"sharon, where is vbp15 on the trial possibility?"
Mar 25

Staff
Sharon Hesterlee posted a blog post

PPMD Awards $100,000 in Exploratory Grants

PPMD is thrilled to award two exploratory grants as part of our ongoing grant program. Peter Arthur of the University of Western Australia was awarded $50,000 to finish testing a molecule called procysteine in animal models in preparation for a human clinical trial. Procysteine is an antioxidant that has been tested in…See More
Mar 25

Staff
Sharon Hesterlee replied to Bernardo A. Iriberri's discussion Ataluren Webinar
"Bernardo, I think they mean substantial risk from an investment standpoint, not due to any issues with the drug.  Because they are a public company they have to be pretty guarded about how "forward looking" their comments are--I think…"
Feb 17

Staff
Sharon Hesterlee replied to Bernardo A. Iriberri's discussion Ataluren Webinar
"Bernardo--here is the recording of the most recent Webinar:  http://community.parentprojectmd.org/events/act-dmd-update-with-ptc-webinar I don't think PTC Therapeutics ever advised the EMA to turn down their application for…"
Feb 17

Staff
Sharon Hesterlee commented on Sharon Hesterlee's blog post Getting There Faster
"Hi Amit--that was interim data.  He is still finishing up the work.  We also funded Dr. Dominic Wells to work with Dr. Ruegg and duplicate the work in his lab.    Sharon"
Dec 2, 2013
amit gupta commented on Sharon Hesterlee's blog post Getting There Faster
"Shron, correct me if I am wrong, but I thought Dr Ruegg presented preclinical report back in April 2013 at the MDA? What do you think is taking so long to present any other report if the preclinical study was complete prior to April?"
Dec 2, 2013

Staff
Sharon Hesterlee commented on Sharon Hesterlee's blog post Getting There Faster
"Hi Amit--I think they're ability to get a trial up and running will depend on having the funding secure and we are still waiting for the final report from the preclinical study.  We'll need to analyze those results to decide if a…"
Dec 2, 2013
amit gupta commented on Sharon Hesterlee's blog post Getting There Faster
"Thanks Sharon. For Tamoxifen - the goal is to have tamoxifen in a clinical trial with Duchenne patients starting in early 2014. Is this still valid?"
Dec 2, 2013

Staff
Sharon Hesterlee favorited Pat Furlong's blog post Grateful for you
Nov 26, 2013

Staff
Sharon Hesterlee posted a blog post

Getting There Faster

PPMD’s research strategy has long had three distinct facets—to develop treatments Better, Faster, and Now. In the past, I have talked about how we have teamed up with TREAT-NMD to use its panel of experts on Duchenne and drug development to review projects with the goal of putting the best…See More
Nov 26, 2013

Staff
Sharon Hesterlee posted a blog post

Who Owns Your Clinical Trial Data?

When parents of children with Duchenne enroll their children in a clinical trial there is often a sense of participating in something bigger than themselves—of course you hope that your child may benefit from a new therapeutic approach, but you also know that all those days of missed school, waiting rooms, endless syringes,…See More
Oct 29, 2013

Staff
Sharon Hesterlee's blog post was featured

World Muscle Society Congress: Next Generation Exon-Skipping & Clinical Trial Updates

The World Muscle Society Meeting in Monterey, California saw reports on a number of topics of interest to the community. On Thursday poster sessions covered advances in the next generation of exon-skipping and updates on various clinical trials.Next Generation Exon-SkippingSeveral laboratories presented data on ways to modify the…See More
Oct 4, 2013

Staff
Sharon Hesterlee posted a blog post

World Muscle Society Congress: Next Generation Exon-Skipping & Clinical Trial Updates

The World Muscle Society Meeting in Monterey, California saw reports on a number of topics of interest to the community. On Thursday poster sessions covered advances in the next generation of exon-skipping and updates on various clinical trials.Next Generation Exon-SkippingSeveral laboratories presented data on ways to modify the…See More
Oct 4, 2013
Ashok Sangwan favorited Sharon Hesterlee's blog post Parent Project Muscular Dystrophy awards $50,000 to Dr. Toshifumi Yokota for multi-exon skipping project
Oct 1, 2013
mehrzad haidari commented on Sharon Hesterlee's blog post DIA Oligonucleotide Conference, Neuromuscular Session Summary
"why Gsk and Prosensa never published the result of dytrophine production level clearly? from 2 years ago that i followed them, i didn't see anything about this important endpoint, but sarepta clearly published the result last year in Phase…"
Sep 27, 2013

Sharon Hesterlee's Blog

PPMD Awards $100,000 in Exploratory Grants

Posted on March 25, 2014 at 11:42am 2 Comments

PPMD is thrilled to award two exploratory grants as part of our ongoing grant program. Peter Arthur of the University of Western Australia was awarded $50,000 to finish testing a molecule called procysteine in animal models in preparation for a human clinical trial. Procysteine is an antioxidant that has…

Continue

Getting There Faster

Posted on November 26, 2013 at 3:45pm 4 Comments

PPMD’s research strategy has long had three distinct facets—to develop treatments Better, Faster, and Now. In the past, I have talked about how we have teamed up with TREAT-NMD to use its panel of experts on Duchenne and drug development to review projects with the goal of putting…

Continue

Who Owns Your Clinical Trial Data?

Posted on October 29, 2013 at 11:00am 0 Comments

When parents of children with Duchenne enroll their children in a clinical trial there is often a sense of participating in something bigger than themselves—of course you hope that your child may benefit from a new therapeutic approach, but you also know that all those days of missed school,…

Continue

World Muscle Society Congress: Next Generation Exon-Skipping & Clinical Trial Updates

Posted on October 4, 2013 at 9:30am 0 Comments

The World Muscle Society Meeting in Monterey, California saw reports on a number of topics of interest to the community. On Thursday poster sessions covered advances in the next generation of exon-skipping and updates on various clinical trials.

Next Generation Exon-Skipping…

Continue

Comment Wall (16 comments)

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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.
 
 
 

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