Novel Ways of Using Immunoglobulin to Treat Disabling Neurological Disorders Are Being Explored

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.

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Yesli’s Story: Income Inequality, Mountainous Terrain and a Shortage of Specialists Hamper Diagnosis and Treatment of Rare Diseases in Chile

By Juan Ambar

June 20, 2017

Yesli Jordán Paillacán overcame a childhood
made difficult by an undiagnosed
primary immunodeficiency.

When we first met Yesli Jordán Paillacán, she was a shy young girl living in Araucanía, Chile’s poorest region in terms of gross domestic product per capita where 80 percent of the population depends on the public health system. Yesli’s life had been continually disrupted by frequent hospitalizations that typically lasted from five to 10 days, and by other health complications that dramatically affected her quality of life.

Access to care in rural areas
She could not attend school regularly, particularly in the winter, when weather conditions exacerbated her condition. Access to care for patients like Yesli in rural areas of Chile is complicated by the distances that often separate them from hospitals and health care centers. In rural areas patients must often travel over rugged mountainous terrain to receive medical treatment.

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The Rarity of Hereditary Angioedema Makes the Disease Especially Dangerous

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.

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Bugs May Be Regaining the Upper Hand Over Once Effective Antibiotics

Antibiotic resistance and rare diseases were among the topics explored by a panel of scientists and pharmaceutical industry representatives in Marburg, Germany.

By Dr. Lutz Bonacker

May 8, 2017

Philipps University of Marburg, Germany, where members of the scientific and pharmaceutical communities examined a range of timely topics.

Earlier this year I joined distinguished members of the scientific and pharmaceutical communities for an academic panel hosted by Philipps University of Marburg, Germany, where CSL Behring operates a manufacturing and R&D facility for recombinant and plasma-derived coagulation factors, C-1 inhibitor and critical care products.

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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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