This Day On The Street
Continue to site right-arrow
This account is pending registration confirmation. Please click on the link within the confirmation email previously sent you to complete registration.
Need a new registration confirmation email? Click here
Stocks Under $10 with 50-100% upside potential - 14 days FREE!

Parent Project Muscular Dystrophy Funds Collection And Analysis Of Imaging Data From Eteplirsen Study

University of Florida to Receive $59,000 Grant from Duchenne-specific Organization

HACKENSACK, N.J., Feb. 1, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) announced that it has funded $59,000 to Krista Vandenborne, PhD  of the University of Florida in Gainesville to test the ability of magnetic resonance imaging  (MRI) to detect changes in the muscles of  those living with Duchenne muscular dystrophy (Duchenne) who participated in the Sarepta phase II trial of Eteplirsen.


"This supplemental funding from PPMD allowed us to very quickly take advantage of a trial in progress to see if MRI can detect changes in the amount and quality of muscle in response to an experimental treatment," said Dr. Vandenborne.

Eteplirsen is an antisense oligonucleotide-based drug that allows skipping of exon 51 in the dystrophin gene.  For approximately 13% of those with Duchenne, skipping exon 51 may lead to the production of a shorter, but functional dystrophin protein.  Sarepta Therapeutics recently completed a phase II study of the drug in 12 participants and found evidence of dystrophin production and some improvement in the distance walked in six minutes.

Although the six minute walk test has been used as a primary outcome in almost all of the current clinical trials for Duchenne, study sponsors are also interested in identifying "biomarkers" or other types of measurements that might indirectly predict which study participants are responding to a therapeutic intervention.  If such biomarkers can be identified and demonstrated to be linked to functional benefits, such as walking ability, they may one day be used in place of functional tests or to add supporting evidence to those tests.

Dr. Vandenborne's group has been using a technique known as "magnetic resonance imaging" or "MRI" to generate pictures of the muscles to demonstrate how much tissue is there, differentiating it from fatty scar tissue.  Due to pilot data generated from an earlier End Duchenne award from PPMD, Dr. Vandenborne received a $7 million grant from the National Institutes of Health to test the ability of MRI to accurately predict the loss of muscle function in boys not undergoing treatment trials. 

Now PPMD has provided an additional $59,000 to Dr. Vandenborne to test the ability of MRI to detect changes in the muscle in response to a treatment – in this case Sarepta's Eteplirsen. The investigators have collected data from the active phase of the study and will continue collecting data as the original study participants stay on the drug in the extended access program.  Results of the MRI analysis from the phase II Eteplirsen study will be submitted for publication as soon as the data is compiled.

"Developing accurate, reliable ways to detect when new drugs are working has become increasingly important, as more drugs enter clinical testing for Duchenne," said PPMD's Founding President and CEO Patricia Furlong.  "This study is part of our ongoing effort to provide tools that will give each new experimental drug the very best chance for success."

This grant was made possible as a result of PPMD's recent holiday campaign which focused on raising funds for projects aimed at identifying biomarkers and genetic modifiers, which will help to unlock better and faster ways of advancing clinical trials.

1 of 2

Select the service that is right for you!

Action Alerts PLUS
Try it NOW

Jim Cramer and Stephanie Link actively manage a real portfolio and reveal their money management tactics while giving advanced notice before every trade.

Product Features:
  • $2.5+ million portfolio
  • Large-cap and dividend focus
  • Intraday trade alerts from Cramer
  • Weekly roundups
TheStreet Quant Ratings
Try it NOW
Only $49.95/yr

Access the tool that DOMINATES the Russell 2000 and the S&P 500.

Product Features:
  • Buy, hold, or sell recommendations for over 4,300 stocks
  • Unlimited research reports on your favorite stocks
  • A custom stock screener
  • Upgrade/downgrade alerts
Stocks Under $10
Try it NOW

David Peltier, uncovers low dollar stocks with extraordinary upside potential that are flying under Wall Street's radar.

Product Features:
  • Model portfolio
  • Stocks trading below $10
  • Intraday trade alerts
  • Weekly roundups
Dividend Stock Advisor
Try it NOW

Jim Cramer's protege, David Peltier, identifies the best of breed dividend stocks that will pay a reliable AND significant income stream.

Product Features:
  • Diversified model portfolio of dividend stocks
  • Alerts when market news affect the portfolio
  • Bi-weekly updates with exact steps to take - BUY, HOLD, SELL
Real Money Pro
Try it NOW

All of Real Money, plus 15 more of Wall Street's sharpest minds delivering actionable trading ideas, a comprehensive look at the market, and fundamental and technical analysis.

Product Features:
  • Real Money + Doug Kass Plus 15 more Wall Street Pros
  • Intraday commentary & news
  • Ultra-actionable trading ideas
Options Profits
Try it NOW

Our options trading pros provide daily market commentary and over 100 monthly option trading ideas and strategies to help you become a well-seasoned trader.

Product Features:
  • 100+ monthly options trading ideas
  • Actionable options commentary & news
  • Real-time trading community
  • Options TV
To begin commenting right away, you can log in below using your Disqus, Facebook, Twitter, OpenID or Yahoo login credentials. Alternatively, you can post a comment as a "guest" just by entering an email address. Your use of the commenting tool is subject to multiple terms of service/use and privacy policies - see here for more details.
Submit an article to us!


DOW 17,778.15 +421.28 2.43%
S&P 500 2,057.40 +44.51 2.21%
NASDAQ 4,743.2230 +98.9110 2.13%

Brokerage Partners

Rates from

  • Mortgage
  • Credit Cards
  • Auto

Free Newsletters from TheStreet

My Subscriptions:

After the Bell

Before the Bell

Booyah! Newsletter

Midday Bell

TheStreet Top 10 Stories

Winners & Losers

Register for Newsletters
Top Rated Stocks Top Rated Funds Top Rated ETFs