“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.
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.
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.
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.
By Joseph Chiao, MD
February 23, 2017
As we look ahead to spring, which is always a busy time for all of us at CSL Behring, we join the hereditary angioedema (HAE) and rare disease communities around the world in celebrating the 10th international Rare Disease Day on February 28. Every year at this time, we proudly join with hundreds of patient organizations worldwide to raise awareness of rare diseases and their impact on people’s lives.
There are upwards of 7,000 rare diseases, with approved treatments for only 5% of these. HAE, a rare and potentially life-threatening genetic condition, occurs in about 1 in 10,000 to 1 in 50,000 people. People with HAE can have considerable swelling episodes referred to as angioedema, which can affect many parts of the body, including the face, abdomen, larynx and extremities.