Joan Miller Advertising Agency

Rare Diseases: How Pharmaceutical Marketers Can Make a Difference

Fast forward and I'm taking her to the hospital every two weeks for casting of a club foot. I keep hoping that her low blood count and other unusual aspects of her birth relate to the foot, but a remark I make about her dimpled knees draws an unexpected response from the resident doing the recasting. He abruptly leaves the room to consult with other doctors. Then Casey is taken away for an X-ray, ultrasound and eventually an MRI. The night of the MRI, her surgeon calls and says that Casey doesn't have kneecaps. He has never seen this before and finds nothing in the medical literature to explain it.

Thus began my life as the mother of a child with a rare disorder.

I immediately went on the Internet, reading every article, study and discussion thread even vaguely associated with my daughter's symptoms. I also did field research, conferring with doctors in 10 hospitals in the Chicago area. I kept reading, thinking it was impossible that Casey could be the only baby on earth with no kneecaps. Then I found a study by a researcher who had identified the genetic marker for a condition called nail-patella syndrome, a rare disorder whose symptoms closely matched my daughter's.

After talking with the researcher at Johns Hopkins, I prepared a detailed brief on our conversation, gathered other materials I'd found on the Internet and presented them to Casey's doctor. That's how we got to a definitive diagnosis, and that's also how helping people with rare diseases became a cornerstone of both my life and my business.

Some basics: Although the Orphan Drug Act defines a rare disease as one that affects fewer than 200,000 people in the U.S., there are approximately 7,000 rare diseases and collectively they affect nearly 30 million people, or one in 10 Americans. Eighty percent are diagnosed in childhood. The category includes cystic fibrosis, hemophilia, all childhood cancers, some forms of muscular dystrophy and numerous less familiar diseases. Rare diseases are often difficult to diagnose, and approximately 70% of treatment therapies -- if they exist at all -- are prescribed off-label.

UniCelebs: Photo Gallery: Christina Hendricks: Critics' Choice TV ...

Actress. Born May 3, 1975, in Knoxville, Tennessee. Christina Hendricks' mother was a psychologist and her father was a British-born forester who worked for the United States Forest Service. Shortly after Hendricks' birth, the family moved to Twin Falls, Idaho, a small, quiet town where one of the few activities available for children was community theater. "My mom got my brother and I involved in a community theater group, so we could create our own fun," Hendricks recalls. "And that's where it all started for me. I spent my whole childhood with this theater called J.U.M.P. Company: Junior Musical Playhouse. It was amazing how many talented kids there were in the group, and also how many people in the community jumped in to volunteer, to direct, to build sets, whatever needed to be done. We'd put on these shows at the community college, and half the town would show up! So it was great. It was the happiest time of my life." When Hendricks was 14 years old, her family moved to Fairfax, Virginia, where she attended Fairfax High School. An artsy kid in a school dominated by jocks and preps, Hendricks struggled to fit in. "I had the worst high school experience ever," she remembers. "People literally spit on me. I was a Goth girl, and in preppy Fairfax County, that did not go over very well. My friends and I were all weird theater people. Some of us were gay. And everyone just hated us." Hendricks dyed her hair purple and wore thick black lipstick. "My mother was mortified and kept telling me how horrible and ugly I looked, and strangers would walk by with a look of shock on their face," she says. "So I never felt pretty, you know. I just always felt awkward." Nevertheless, no amount of hair dye or dark makeup could obscure Hendricks' increasingly apparent beauty, and toward the end of high school a friend persuaded her to enter a modeling competition to appear on the cover of Seventeen magazine. "When I got the pictures back my mum and I started crying because I had no idea I could be pretty," she recalls. "It just sort of changed my perspective of myself." Hendricks did not win the contest but at the age of 19, after graduating high school, she moved to New York and began modeling professionally. Hendricks thoroughly enjoyed her time as a model. "I loved it a lot," she says. "I know a lot of girls who are like, 'Ugh, I hated it,' and I'm like, 'Really? You hated being paid to travel all around the world and have your picture taken?

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