August 23, 2016
By Toby Simon, MD
Zika virus has been widely reported in the news during the past year, so much so that a number of athletes chose to stay home rather than compete in the Olympics in Rio de Janeiro and risk infection. We therefore understand that patients treated with our plasma derived products want to be certain they are not at risk for infection with Zika virus through their treatment with our products.
Infection with the Zika virus generally results in no symptoms, but 20% or more of individuals infected will have a mild illness of 5 to 7 days with fever, rash, headache, and discomfort in their hands, wrists, and ankles. This virus has also been associated with Guillain-Barre syndrome, a self-limited disease of the nervous system that can be serious.
Most of the public health concern relates to infection in pregnant women, because microcephaly (incomplete brain development) and other fetal defects have resulted from infection with this virus. The virus is spread by mosquito bites, but there is evidence of transmission through sexual contact and blood transfusion. The virus has been most common in tropical areas. The epidemic is severe in Puerto Rico and has emerged in the United States through both travel and in the last month from local mosquito transmissions in south Florida.
“Part of me was slightly ashamed that I had hemophilia growing up. It was something I tried to hide.”
August 4, 2016
New father and hemophilia A patient Tim Grams is an outdoor adventurer. See how Tim isn’t letting this hereditary blood disorder keep him from enjoying the Colorado outdoors, including hiking Dinosaur Ridge and mountain biking at Red Rocks Park. Click the image below to watch the video.
The science of pharmacometrics is changing the way some therapeutic recommendations are expedited around the world.
By Jagdev Sidhu, PhD and Michael Tortorici, PhD
At CSL Behring, we have a keen understanding of the difference that accelerated access to vital therapies as well as therapeutic recommendations can mean for patients with rare and serious medical disorders.
Pharmacometrics offers an exciting alternative to traditional clinical trials in certain instances by allowing the approval of more flexible usage of therapeutic products without a clinical trial. For the majority of patients with a rare disease who are on chronic therapies, this is good news indeed.
The approval of medicines and their therapeutic options takes place in a rigorous and uncompromising regulatory environment in which the wellbeing of patients is the main priority. At the same time, regulators around the world are increasingly engaging with the pharmaceutical companies to advance methodologies, such as the use of pharmacometrics that bring innovation to the drug development process.
By Iris Jacobs, MD
It’s difficult for most of us to fully appreciate what it’s like to have a rare disease caused by a deficient or defective protein. Traditionally, patients with certain types of protein deficiency or defect had no option other than to be intravenously injected two to three times per week with conventional therapies.
Teamwork: bringing rIX-FP to patients is a culmination of several years of work across the entire global organization. Seen here are members of CSL Behring’s rIX-FP Clinical Research and Development team in King of Prussia, Pa., U.S.
While frequent injections (prophylaxis) may prevent the onset of symptoms in most instances, this approach is a burden for patients who lead busy lives. It is also the main reason that routine prophylaxis is often delayed in young children. In addition, some symptoms can cause progressive and in certain patients debilitating damage.
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.