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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Women with Bleeding Disorders – Are they Often Overlooked?

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.

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Is Unnecessary Paperwork and Documentation
Putting Patients at Risk?

By Dina Inverso

March 15, 2017

Managing the cost of prescription drugs is a major economic challenge. As a result, prior authorization requirements are more demanding than ever. And, for doctors and patients, navigating the maze of regulations and restrictions can be frustrating at best. At worst, patient access to lifesaving therapies sometimes hangs in the balance.

A paradigm shift is required for people with rare medical conditions who require chronic care with medicines that are life-sustaining. This is a cost for life, and payers should focus on lifetime management cost. But instead, from the reimbursement coverage perspective, the process has become more granular and we are seeing more and more hurdles being created.

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Without Human Plasma, Many Patients with Rare and Serious Medical Disorders Would Not Have Treatments Available to Them

That’s why plasma donors are heroes to millions of patients who receive plasma-derived therapies.

October ­­7, 2016
By Daniel Ferris

Barb and Rusty Roberts

Immune deficiency patient Barb Roberts with Rusty Roberts vacationing in Dinard, France.

Every autumn the plasma protein therapeutics industry recognizes the lifesaving contributions of plasma donors during International Plasma Awareness Week (IPAW).

At CSL Behring and CSL Plasma, we appreciate the critical role donors play in ensuring a reliable and secure source of human plasma as worldwide demand continues to rise. We value their donations not just during IPAW (October 9-15), but year-round.

This is because plasma-derived therapies are crucial for patients’ well-being and health, and an appropriate supply of products is absolutely essential and depends on a sufficient supply of collected plasma.  

While it can take more than 1,000 plasma donations to treat one adult patient for a year, the current collection system allows for an appropriate supply of product.
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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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