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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 this discussion. Last reply by Sharon Hesterlee Sep 8. 2 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 replied to Moein's discussion Pfizer Myostatin Inhibitor&HT-100
"Sorry David, I really can't speculate.  If Pfizer hasn't made something public its generally because they are not yet ready to talk about it. Sharon"
22 hours ago

Staff
Sharon Hesterlee replied to Moein's discussion Pfizer Myostatin Inhibitor&HT-100
"Moein, I don't believe the company has released any results from the healthy volunteer study.  Sharon"
yesterday

Staff
Sharon Hesterlee replied to Moein's discussion Pfizer Myostatin Inhibitor&HT-100
"Hi Moein:  The Pfizer drug is different from the ACE 031 drug--it is more specific in it's activity and you might predict that it's less likely there will be the kind of side effects that were seen in the ACE 031 trial, but…"
yesterday
Moein favorited Sharon Hesterlee's profile
yesterday

Staff
Sharon Hesterlee replied to Moein's discussion Pfizer Myostatin Inhibitor&HT-100
"Andrew, I believe that a Duchenne trial is planned and hopefully we will hear something about that soon. Sharon"
yesterday

Staff
Sharon Hesterlee replied to Moein's discussion Pfizer Myostatin Inhibitor&HT-100
"Just to clarify some details, the Pfizer drug is not the same drug that tested previously by Acceleron and Shire (ACE-031) or Wyeth's MYO-029.  This is a completely new drug that has never been tested in muscular dystrophy before and works…"
yesterday

Staff
Sharon Hesterlee replied to Kenny Chuck's discussion Follistatin Gene Therapy
"Kenny, here's a link to Dr. Mendell's presentation at the conference this summer: http://videos.parentprojectmd.org/conference14/audio/03_Saturday/S05_Mendell.mp3 "
Oct 23

Staff
Sharon Hesterlee replied to Sharon Hesterlee's discussion Japanese company to start exon-53 skipping trial (morpholino)
"Hi Alexandre-- We've had some difficulties tracking down a contact at this company but we are meeting with the Japanese Ministry of Health in October and this project is on my short list of things to ask about--will post an update here after…"
Sep 8
Alexandre da Costa e Silva replied to Sharon Hesterlee's discussion Japanese company to start exon-53 skipping trial (morpholino)
"Hi Sharon. My name is Alexandre and my son Rafael has deletion of Exons 48-52. Do you have any news about this Japanese study? Thank you."
Sep 8

Staff
Sharon Hesterlee replied to Dawn Jones's discussion still waiting on official diagnosis
"Hi Dawn: So ataluren (new brand name Translarna) has been approved in Europe but I don't think even the Europeans have access to it yet because each company's government has to decide if they are going to pay for it.  I don't…"
Sep 2

Staff
Sharon Hesterlee replied to Dawn Jones's discussion still waiting on official diagnosis
"Dawn, Janine is right, Ann Martin (ann@parentprojectmd.org) at PPMD is a licensed genetic counselor who can help you interpret the results.  Although the diagnosis is never a good thing, a premature stop codon mutation may give him several…"
Sep 2
Satish left a comment for Sharon Hesterlee
"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"
Jul 23

Staff
Sharon Hesterlee commented on Sharon Hesterlee's blog post No Exon Left Behind
"Hi Kimmy:  Neither Prosensa nor Sarepta are working on an exon 43 skip.  If you want to email me privately and tell me what your son's deletion is, I would be happy to look at the options.  Sharon@parentprojectmd.org. Sharon"
Jul 22
kimmy watters commented on Sharon Hesterlee's blog post No Exon Left Behind
" hi sharon i have question  exon skipping   43  are the drug company  began  trails  in  jan 2015"
Jul 22

Staff
Sharon Hesterlee commented on Sharon Hesterlee's blog post No Exon Left Behind
"David:  Prosensa is using the mouse to work out the details for a multi-exon skip that has never been demonstrated before (for these particular exons).  They are also continuing to move forward several clinical trials for single…"
Jul 22

Staff
Sharon Hesterlee commented on Sharon Hesterlee's blog post No Exon Left Behind
"Hi Rupjani: Exon-skipping is certainly not the only game in town and it's not our major focus as an organization.  We deliberately have a very diversified portfolio when it comes to therapy development because you really never know which…"
Jul 22

Sharon Hesterlee's Blog

No Exon Left Behind

Posted on July 21, 2014 at 10:15am 6 Comments

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…

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New Duchenne Therapies in Development

Posted on April 24, 2014 at 12:30pm 3 Comments

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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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…

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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…

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Comment Wall (17 comments)

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At 2:18pm on July 23, 2014, Satish said…

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

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?

 
 
 

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