Bright Future for Rare Disease Patients Driven by Innovations Including Genomics and Personalized Medicine, But Some Challenges Remain

International Bio Convention

CSL Behring’s Bob Repella joined NORD’s Pam Gavin for a wide-ranging discussion about rare diseases at the 2016 BIO International Convention. The following is excerpted from his remarks.

By Bob Repella

Rare diseases are serious and often debilitating conditions that have a tremendous impact on 350 million patients worldwide, about half of whom are children, and their families. Today we are on the brink of significantly improving their lives through rare disease awareness, early diagnosis and innovative treatments.

More therapies are available today for previously unmet needs, and there are greater choices for patients than ever before. But even with this progress, a great deal of work remains to be done. Over 90% of the approximately 7,000 rare diseases have no treatment available, and there are still challenges to accessing existing therapies. Nevertheless, the trends overall are positive.

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100 Years Old and Just Getting Started!

Centenary Logo

April 25, 2016
By Paul Perreault

Today we proudly mark CSL’s 100th anniversary, a milestone that is a testament to our values, science, people, and the patients we serve. For the past 100 years our vision, focus and agility have remained steadfast, enabling us to accomplish this feat. We have a rich heritage with an even brighter future. In many ways, we’re just getting started.

As a tangible expression of our values in action, and as a way of celebrating the CSL Centenary, we are establishing a $25 million R&D Fellowship program for early stage and translational research. The R&D Fellowship reflects the promise we made a century ago to save lives and protect people’s health.
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Rare Disease Day Focuses International Attention on the Rare Disease Community

Rare diseases are an important public health issue.

rare-disease-day-2015

February 12, 2015

By Peter L. Saltonstall

Each year since 2009, the National Organization for Rare Disorders (NORD) has been the official U.S. sponsor of Rare Disease Day®1. This is a day celebrated around the world that is dedicated to elevating public understanding of rare diseases and to calling attention to the special challenges faced by the rare disease community.
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