That’s why awareness opportunities such as HAE Day are so important.
By Debra Bensen-Kennedy, MD
May 15, 2017
Like many physicians, I learned about hereditary angioedema (HAE) during a brief moment in my years of education and training. Had I not worked at CSL Behring, HAE would likely never cross my mind again, given its rarity.
Medical professionals juggle troves of clinical information, and on HAE Day we help keep the disease top of mind. Because HAE attacks can be life-threatening if they occur in the throat, it is extremely important for healthcare professionals to know what to do when they encounter a patient, even if their only other experience with the condition was during their medical training.
The answer is ‘yes,’ which is why World Hemophilia Day 2017 is raising awareness of the many women with bleeding disorders who often go overlooked and undiagnosed.
By Jerry Powell, MD
April 13, 2017
No other genetic disease has made as much progress as hemophilia over the past half century. The typical life expectancy for boys born with severe hemophilia has increased from approximately 20 years to near normal life expectancy. One reason for this tremendous progress has been the involvement of patients and their families in supporting research and development (R&D) and participation in clinical trials, which has brought new innovative treatments to market.
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.
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
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.