The Challenges of Diagnosing, Treating and Finding a Cure for Primary Immunodeficiency – One Doctor’s Perspective

More patients are being properly diagnosed and treated than ever before, but obstacles remain in the pursuit of a cure.

By John Sleasman, MD

April 27, 2017

Immunologists are using innovative treatments such as cell-based therapies at Duke Children’s Hospital and Health Center (pictured above), which involves replacing missing cells or genes to correct primary immune deficiency diseases.

Photo courtesy of Duke Health

This is an exciting time in the Department of Pediatrics at Duke School of Medicine’s Division of Allergy and Immunology. Late last year we opened the Jeffrey Modell Center (JMF) for research and clinical care for children with primary immunodeficiency (PI) diseases — a group of more than 300 rare, chronic disorders in which part of the body’s immune system is missing or functions improperly.

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What Will Healthcare Look Like in 50 to 100 Years?

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.

BioFest 2016

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.

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Coagulation Research Award Taps Brain Power and Vision of New Generation of Researchers

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.

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Lifesaving Medicines Should Never Be in Short Supply

That’s why we’re building for the future in Lengnau and around the world to meet growing demand and bring more therapies to more patients.

September 22, 2016
By Uwe E. Jocham

Lengnau Construction

These are exciting times at CSL Behring, as we expand our infrastructure to keep pace with the increasing demand for new innovations that address unmet medical needs or enhance current treatments.

We are currently building for the future in Lengnau, Switzerland, where we are constructing a state-of-the-art recombinant manufacturing facility. The foundation stone of the new structure was laid in May. With this production site, CSL Behring is creating a new product family based on state-of-the-art technology.

The plant will manufacture and globally market three new recombinant coagulation factors: recombinant Factor IX Fusion Protein for patients with hemophilia B, recombinant Factor VIII-Single Chain to treat hemophilia A, and a recombinant Factor VIIa-Fusion Protein treat hemophilia patients with inhibitors.
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Are Clinical Trials Always Necessary?

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.

Main Frame

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.

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