This Day On The Street
Continue to site right-arrow
ADVERTISEMENT
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
$1 buys you full access to ALL of TheStreet's Subscription Services! Learn More

Alexion Joins Patient Organizations Worldwide In Recognizing Rare Disease Day 2014

Stocks in this article: ALXN

Alexion Pharmaceuticals (Nasdaq:ALXN) joins the European Organization for Rare Diseases (EURORDIS), the National Organization for Rare Disorders (NORD) and patient organizations worldwide in recognizing Rare Disease Day 2014, a global effort to increase awareness of rare diseases, their profound impact on patients and the need for improved diagnosis and treatment.

Alexion supports key objectives of EURORDIS, NORD and other patient organizations through its mission to develop and deliver life-transforming therapies for patients worldwide who suffer from severe and life-threatening rare diseases.

Diagnosis and Treatment Challenges for Rare and Ultra-Rare Diseases

Many rare and ultra-rare diseases are chronic, progressive and marked by continuing pain, severe disability and high mortality. Few physicians are familiar with diagnosing and treating these illnesses, which frequently leads to missed, delayed or inaccurate diagnoses even when an approved, effective therapy is available. 1 According to global survey results published in the current issue of The Journal of Rare Disorders (JRD), conducted on behalf of Global Genes, patients living with rare diseases visited an average of 7.3 physicians before receiving an accurate diagnosis. 2 The survey also reveals that 44 percent of patients surveyed believed that a slow diagnosis resulted in delayed treatment with a negative impact on their condition. 2

“Patients with rare and ultra-rare life-threatening disorders often face long delays in receiving an accurate diagnosis. Without an accurate diagnosis, or access to effective treatment options, patients too often are at risk for catastrophic medical consequences,” said Leonard Bell, M.D., Chief Executive Officer of Alexion. “On this Rare Disease Day and every day, through our continued commitment to breakthrough medical research, each of us at Alexion are focused on improving the knowledge of rare diseases and developing and delivering therapies to transform the lives of these patients worldwide.”

Delivering Life-Transforming Therapies Across the Globe

Alexion’s research and development programs are focused on highly innovative therapies with the potential to transform the lives of patients with severe and life-threatening ultra-rare disorders and for which there are no effective treatment options. The company’s development programs are solely focused on:

  • Severe disorders with devastating and life-threatening medical consequences
  • Disorders with ineffective, or no treatment options
  • Disorders that are ultra-rare and affect very small numbers of patients

Scientists at Alexion are working relentlessly to understand the underlying causes of these diseases and to discover and develop breakthrough medicines to treat them. To learn more about Alexion’s Research & Development programs, visit www.alexionpharma.com/pipeline.

To learn more about Rare Disease Day, visit www.rarediseaseday.us for U.S. activities and www.rarediseaseday.org for global activities.

About Rare and Ultra-Rare Disorders

In the United States, a disease is defined as rare if it affects fewer than 650 patients per million of population. 3 The European Union definition of a rare disease is one that affects fewer than five patients per 10,000 of population. 4 In contrast, a disease is generally considered to be ultra-rare if it affects fewer than 20 patients per million of population 5 (one patient per 50,000) – and most ultra-rare diseases affect far fewer people than this.

Despite the very small numbers of patients they affect, the impact of these rare and ultra-rare diseases on patients, their families, and society is profound, as many of these conditions are severe, chronic and progressive, with significant premature mortality. Patients with severe and life-threatening ultra-rare diseases often live without hope, have no effective treatment options and may face premature death.

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!

Markets

DOW 17,827.75 +12.81 0.07%
S&P 500 2,072.83 +5.80 0.28%
NASDAQ 4,787.3170 +29.0650 0.61%

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