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!

Joint Program In Spinal Muscular Atrophy Enters First Stage Of Clinical Development

SOUTH PLAINFIELD, N.J., Jan. 22, 2014 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT), the SMA Foundation, and Roche (SIX: RO, ROG; OTCQX: RHHBY), announced today that their joint research program in Spinal Muscular Atrophy (SMA) has entered the first stage of clinical development aiming to assess safety and tolerability of an oral compound in healthy volunteers. SMA is a genetic neuromuscular disorder that leads to muscle weakness and mobility impairment and is considered the leading genetic cause of death in infants and toddlers.   

(Logo: http://photos.prnewswire.com/prnh/20010919/PTCLOGO)

Luca Santarelli, Head of Neuroscience, Ophthalmology and Rare Diseases at Roche commented: "The compelling science behind this project and the highly synergistic alliance among Roche, PTC Therapeutics and the SMA Foundation are the groundwork for this research program. Although still early in development, we consider every step towards a therapeutic option in SMA of high importance for patients and families affected by this devastating and currently untreatable condition."

SMA is caused by a missing or defective SMN1 gene, which results in reduced levels of the survival motor neuron (SMN) protein. SMN protein plays a critical role in the survival of neuronal cells in the spinal cord responsible for muscle movements. Reduced levels of SMN protein lead to dysfunction and death of the neuronal cells, making muscles smaller and weaker over time, and can even result in death in the most severe forms of the disease. The orally available small molecule compounds in the program are designed to target the underlying cause of the disease by increasing SMN protein levels in the nervous system, muscles, and other tissues. It is estimated that approximately one in 10,000 children are born with this rare genetic disorder.

"We are delighted with the achievement of this important milestone," commented Stuart W. Peltz, Ph.D., CEO of PTC Therapeutics, Inc. "The innovative approach developed at PTC has benefited tremendously from the scientific partnership and dedication of both Roche and the SMA Foundation. The rapid progress highlights our combined dedication to advance an investigational medicine for SMA as rapidly as possible."

The SMA program was initiated in 2006 by PTC Therapeutics in collaboration with the SMA Foundation, which provided the majority of its funding prior to Roche joining the collaboration.

1 of 2

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 16,912.11 -70.48 -0.42%
S&P 500 1,969.95 -8.96 -0.45%
NASDAQ 4,442.6980 -2.2110 -0.05%

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