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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World hae day :-) – Many Faces, One Family

Hereditary Angioedema patients are a global family tied together by the need to reduce the time it takes to diagnose and treat this rare disease.

By Henrik Balle Boysen

World PI Week

Hereditary Angioedema International (HAEi) is a non-profit global network of national hereditary angioedema (HAE) patient associations for C1-Inhibitor deficiencies. We are dedicated to raising awareness of HAE and the people it affects around the world.

We strive to improve the time it takes to diagnose HAE and facilitate access to and reimbursement for life saving HAE therapies, which will enable lifelong health for all patients no matter where they live.

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Alice’s Story

Anyone coping with a rare disease knows firsthand how difficult and frustrating it can be to find the correct diagnosis and proper treatment.

February 3, 2016

At CSL Behring, we understand the unique challenges faced by people stricken with life-threatening medical conditions because of our long experience, deep knowledge and unwavering focus on preventing and treating serious diseases.

Using the latest technologies, we are unlocking the potential of biotherapies to save lives and to help improve the quality of life of people living with rare and serious medical conditions. Our operational excellence combined with our focused global R&D organization enables us to efficiently identify, develop and deliver innovations that patients need and want.

We’ve never been more excited about emerging new medicines and technologies that hold the key to unprecedented opportunities to improve patient well-being. But the reality is that it can still take years just to obtain an accurate diagnosis.

The good news is that when the search is over, many people like Alice can – and do – lead active, fulfilling lives. This is her story.

Why Aren’t More Patients Able to Infuse Immunoglobulin at Home?

April 21, 2015

By Lawrence A. LaMotte
World PI Week
Allowing patients to receive their immunoglobulin infusion in the comfort of their own home on a schedule that meets their needs has a significant impact on quality of life. This is particularly true for patients who must travel long distances to and from their health care provider. Home infusion may also be more prudent for patients with a primary immunodeficiency (PI), who are especially vulnerable to infections acquired in a hospital setting.
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