Researchers are targeting autoimmune epilepsy and chronic inflammatory demyelinating polyneuropathy.
By Karen MacPhail and Gabriela Espinoza, MD
July 25th, 2017
As many as one billion people worldwide suffer from more than 600 neurological disorders, according to the World Health Organization and University of California, San Francisco, respectively. Of that number, it is estimated that 6.8 million people die each year from neurological disorders ranging from epilepsy to Alzheimer’s disease.
Neurological disorders affect the central nervous systems or the peripheral nervous systems and can impair the brain, spinal cord, peripheral nerve or neuromuscular function.
If we focus on innovation and sustainability, healthcare will evolve in ways that are in sync with the changing needs of patients, the economy and global politics.
The following post is excerpted from Paul Perreault’s remarks at International BioFest 2016 in Melbourne, Australia.
December 6, 2016
Healthcare statisticians tell us that the first person who will live to 150 years of age has already been born. Considering human biology and the challenges we would need to overcome, doubling our life expectancy at first seems fanciful. But with our understanding of science and the refined art of personalized medicine, 150 may be reasonable.
In fact, our understanding of human biology is expanding at an exponential rate. Ten years ago it took the world’s greatest minds 10 years to map the human genome. Now it takes one day. Even for those of us in the healthcare sector, it is virtually impossible to predict what healthcare will look like in 50 or 100 years. But there are constants to guide us.
The CSL Behring Professor Heimburger Award is helping to ensure young researchers around the world have the opportunity to bring their innovative ideas to the forefront.
October 17, 2016
We are seeing some exciting research as CSL Behring delivers on our promise to advance research and innovation through grants such as the Heimburger Award.
This is particularly important for young researchers working in clinics who often don’t have time to raise money for their research. Adding to the challenge is the fact that government funding of orphan disease research is often difficult to obtain.
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.
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.
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