"Raktim: I think this is a great idea! I get so many emails from India about the best local resources and local research. I know some of the investigators in India, but it would be great if I could direct people here to…"
Staff
-
Sharon Hesterlee
- Female
- Tucson, AZ
- United States
- Featured Blog (8)
- Discussions (102)
- Events
- Groups (2)
- Photos
- Photo Albums
- Videos
Sharon Hesterlee's Groups
Sharon Hesterlee's Discussions
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
FDA Hearing on Rare Disease Drug Evaluation--Comment Period
Started this discussion. Last reply by Sharon Hesterlee Jul 8, 2010. 5 Replies 0 Favorites
The Food and Drug Administration is holding a public hearing on June 29th called "Considerations regarding FDA review and regulation for Articles for the treatment of rare disease." I've attached…Continue
Research Info on the PPMD site
Started this discussion. Last reply by Terry Porcaro Feb 23, 2010. 5 Replies 0 Favorites
So, I'm hoping I can get some feedback from this group (feel sure that I will!) on changes to the research information on the PPMD Web site. What I envision is a detailed graphic that charts all of…Continue
Sharon Hesterlee's Page
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 and State:
- Tucson, AZ
- Country:
- US
Latest Activity
kimmy watters left a comment for Sharon Hesterlee" 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…"
StaffSharon Hesterlee replied to Donna Cicardo's discussion Will there ever be studies other than exon skipping and such????
"
Hi Amit:
Good point--I think I was thinking of the first Summit Study that would include boys with DMD, which could potentially take place in 2013 if the safety study planned for this year is positive (and on time), but the…"
StaffSharon Hesterlee replied to Donna Cicardo's discussion Will there ever be studies other than exon skipping and such????
"Hi Donna:
I think you would have to skip exons 2-7 to put an exon 2 deletion back into frame--I'm not sure that we can do that big of a skip yet. I'm not sure what is meant by that Web site.
Sharon
"
StaffSharon Hesterlee replied to Donna Cicardo's discussion Will there ever be studies other than exon skipping and such????
"Hi Donna:
There are a lot of drugs in testing and development that don't depend on exon-skipping. Some approved drugs like Viagra and Spironolactone may end up working as well or better than some of these high tech approaches--the jury is…"
StaffSharon Hesterlee replied to Rupjani B's discussion Project Catalyst : what is the present status of their utrophin upregulation drug candidate
"Mission accomplished, lol! I do lose considerable sleep over these deadlines.
And I'm pretty sure that Pat never sleeps at all. I think the only thing I would take issue with is the idea that you can control the time lines if…"
StaffSharon Hesterlee replied to Rupjani B's discussion Project Catalyst : what is the present status of their utrophin upregulation drug candidate
"
David, I absolutely agree that on the whole there is too much acceptance of status quo for timelines and regulatory requirements etc. PPMD has focused its research efforts on activities specifically designed to speed up the pace of…"
StaffSharon Hesterlee replied to Rupjani B's discussion Project Catalyst : what is the present status of their utrophin upregulation drug candidate
"
Hmm..so David. Not to be difficult, but aren't the Secklers raising money for this project through donations? I know they bought the IP, but after that it becomes another industry drug development project--Halo…"
StaffSharon Hesterlee replied to Rupjani B's discussion Project Catalyst : what is the present status of their utrophin upregulation drug candidate
"Not sure yet, actually.
Sharon"
StaffSharon Hesterlee replied to Rupjani B's discussion Project Catalyst : what is the present status of their utrophin upregulation drug candidate
"Hi Rupjani:
The Project Catalyst utrophin upregulator probably won't go to trial until 2013. I know this has been a really long road--I think we first gave them funding to do the original drug screen in 2004. We did a Webinar…"
StaffSharon Hesterlee replied to Tulika's discussion IS this the large animal study that muscle regeneration was waiting for
"
Hi Tulika:
So, the stem cell workshop that I mentioned in my post above is taking place in June in conjunction with the New Directions in Muscle Biology and Disease meeting in New Orleans (it is funded by PPMD). if…"
StaffSharon Hesterlee replied to Tulika's discussion One year on Phase 2 of GSX/Prosensa Trial
"
So, we are actually getting beautiful and current natural history data from the placebo arms of all these trials--if we could aggregate that data we would have a large sample size. There is some precedent for doing this kind of…"
Sharon Hesterlee's Blog
World Muscle Society 2011 Report
Posted on November 8, 2011 at 9:30am 4 Comments 1 Favorite
At the recent World Muscle Society meeting, which took place in Portugal, a great deal of new data on exon skipping was presented, both on the status of current human clinical trials and on the efforts of investigators to improve the efficiency of the technique by combining exon-skipping with gene and stem cell delivery. Below are a few highlights from the meeting with the caveat that it was impossible to cover every poster and talk.
Muscle Stem Cells
Jenny…
ContinueOut and About
Posted on April 7, 2011 at 2:24pm 14 Comments 0 Favorites
I’ve just spent two weeks in a row on the road at different meetings—first the Health Research Alliance (HRA) meeting in New York and then the Muscular Dystrophy Association meeting in Las Vegas.
HRA is an organization made up of most of the major non-profit funders of health research -- the members meetings are designed to keep us up to date on the latest trends in research and funding and to allow us to learn from one another. I chair the HRA Working Group on “Funders of…
ContinueDr. Rhodes -- STS
Posted on November 10, 2010 at 10:57am 20 Comments 0 Favorites
I spoke at length last week with Dr. Rhodes about ways that we could better determine the effectiveness for DMD of his VECTTOR electrical stimulation system, which Dr. Rhodes says stimulates acupuncture points, reflexology points and free nerve endings. Dr. Rhodes was very up front about the fact that he isn’t a scientist and isn’t an expert on DMD and his actual professional training is in podiatry. He has supplied his VECTTOR machine to 36 families with muscular dystrophy—he didn’t specify… Continue
Beyond Exon 51 in the Land Down Under
Posted on September 20, 2010 at 8:17pm 33 Comments 0 Favorites
There’s often a fine line between “out-of-the–box” and “out-in-left-field.” I guess when my blog about Duchenne research starts with that statement you know I’m probably going to come down one way or another about something, but it might not be the direction you’re expecting.
This community lives with a lot of hope and hype. Many of you have learned that when you hear something too good to be true that it’s probably not. You know the principles of scientific experimentation, you… Continue
This community lives with a lot of hope and hype. Many of you have learned that when you hear something too good to be true that it’s probably not. You know the principles of scientific experimentation, you… Continue
Comment Wall (16 comments)
- 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.
Need help using this community site? Visit Ning's Help Page.
 |
 |
 |
Events
-
Paint for Peyton to end Duchenne
June 8, 2012 from 6:30pm to 7:30pm – Spirited Art
0 Comments 0 Favorites
© 2012 Created by PPMD.
