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
TheStreet Open House

Repligen Reports Positive Results From Phase 1 Clinical Trial Of RG3039 For Spinal Muscular Atrophy

In addition to the presentation at AAN, Dr. David Jacoby, Medical Director at Repligen Corporation, will present the RG3039 Phase 1 study outcomes during the 2012 International SMA Research Group Meeting. This FSMA organized meeting is being held in Minneapolis from June 21-23, 2012. Dr. Jacoby’s presentation titled, “A Phase I Study in Healthy Volunteers to Assess the Safety, Pharmacokinetics and Pharmacodynamics of the DcpS inhibitor RG3039,” is scheduled for Friday, June 22, 2012 at 3:40 p.m.

About Spinal Muscular Atrophy

Spinal muscular atrophy is an autosomal recessive neuromuscular disease in which a defect in the SMN1 (survival motor neuron) gene results in low levels of the protein SMN and leads to progressive damage to motor neurons. It is the leading cause of infant mortality and the second most common inherited neuromuscular disease, with symptoms that typically emerge before the age of two. SMA is characterized by progressive muscle weakness leading to severe physical disability and often, loss of life due to respiratory insufficiency. The prevalence of SMA in the U.S. and EU is approximately 20,000 patients.

Orphan Drug and Fast Track Designations

Repligen has previously received U.S. Orphan Drug and Fast Track designations for RG3039 for the treatment of SMA, as well as Orphan Medicinal Product designation in the EU. Fast Track is a process designed to facilitate the development and expedite the review of drugs that have the potential to treat serious disease and fill an unmet medical need. The orphan programs provide incentives for the research, development and marketing of products intended to diagnose, prevent or treat rare conditions and/or serious or debilitating diseases with unmet medical needs. Orphan designation grants the sponsor exclusive marketing rights for seven years in the U.S. and ten years in the EU following regulatory approval of the designated product. In the U.S., rare diseases are defined as those affecting fewer than 200,000 Americans. In the U.S., rare diseases are defined as those affecting fewer than 200,000 Americans. In the EU, rare diseases are considered those that affect no more than five per ten thousand persons in the community.

3 of 5

Select the service that is right for you!

COMPARE ALL SERVICES
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 17,131.97 +100.83 0.59%
S&P 500 1,998.98 +14.85 0.75%
NASDAQ 4,552.7590 +33.8570 0.75%

Brokerage Partners

Rates from Bankrate.com

  • 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