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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Baylee’s Story

“It’s a miracle you’re still alive.”

April 20, 2017

High school student and common variable immune deficiency (CVID) patient, Baylee Gregory, is an avid golfer and soccer player who hopes to one day play those sports in college. That didn’t seem possible years ago when a doctor commented, “It’s a miracle you’re still alive.”

Now that she’s been diagnosed and is treating her primary immunodeficiency, her mom, Annette, says, “She’s going to live a life that she wants to live and not let things stand in her way.” Baylee and her mom shared their story with BioBlog at the start of World PI Week 2017.

BAYLEE: I always had sinus infections. I always had ear problems, stomach issues. Family trips I would always be the one sick. I’d go to the doctor and they would just tell me that it was allergies or take an antibiotic, it’s another sinus infection, it’ll go away.
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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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From Scrappy Beginnings, an Industry Leader That
Hasn’t Forgotten Its Roots

By Paul Perreault

March 7, 2017

One of the things I like to remind our people to do is remember where we came from. In reflecting on my recent interview with Medical Marketing & Media (MM&M) magazine, in which I talked about CSL Limited’s unique beginning as a government agency, it strikes me that we really are unlike other biotechs.

Founded by the Australian government in 1916, CSL was born to serve people. It wasn’t until 1994 that we were privatized. CSL was established to ensure a continuous supply of medicines to Australians would not be disrupted by World War I. As I mentioned in my interview, we started with a mindset that reflected our government heritage – we’re always scrapping.

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