Pixel Tracker

Tuesday, March 19 , 2019, 9:37 pm | Light Rain 52º

 
 
 
 

Karen Telleen-Lawton: A ‘Rare’ Day to Show Your Stripes

What do zebras and Feb. 29 have in common? Zebras are rare among equines for their black and white stripe pattern. Feb. 29ths are not strictly rare, but certainly noteworthy: They come along only every 1,460 days. These two phenomena combine on the last day of February to commemorate Rare Disease Day.

You can show your support for people with rare diseases and rare disease research by “showing your stripes” and by posting to social media.

A rare disease is defined in the United States as one that afflicts fewer than 200,000 Americans at any time, according to the National Organization for Rare Disorders. In Europe, the definition is a disease affecting no more than one in 2,000 people. Incredibly, there are 7,000 such diseases, prompting a saying in the field, “While a rare disease is rare, rare diseases are common.”

Rare Disease Day began in 2008. This year, events will be held in more than 90 countries.

The history of focusing on rare diseases for recognition and research began with the infamous drug thalidomide. Prescribed in the 1950s and 1960s to alleviate pregnant women’s morning sickness, the medication caused malformation of fetus’ limbs and some organs. The drug resulted in a 40 percent mortality rate of children whose mothers had taken it.

Thalidomide’s devastation led to much stricter drug development requirements. Drug manufacturers subsequently focused on common diseases, where the large number of potential patients would increase the likelihood of a new drug’s profitability. Rare diseases were “orphaned” by this chain of events, languishing with little or no research.

In December 1981, Rep. Henry Waxman, D-CA, presented a bill in Congress called the Orphan Drug Act. The bill called for incentives for drug companies to develop drugs for rare diseases. Incentives included tax breaks, enhanced patent protection and marketing rights, clinical research subsidies, and the creation of a government-run enterprise to engage in research and development. The Orphan Drug Act became law in January 1983.

Drug companies are nearly unanimous in declaring the success of ODA. Critics argue that some of the drugs developed under the act were in the queue anyway. Nevertheless, statistics show an amazing difference. In the decade previous to 1983, fewer than 10 such drugs came on the market in the United States. In the two decades after, more than 1,100 orphan drug designations were granted and about 250 orphan drugs received marketing authorization.

This research has resulted in treatment for 200 rare diseases thus far. In just the past few years, new medicines help patients with ailments such as cystic fibrosis, several enzyme deficiency disorders and more than a dozen new cancer therapies. Furthermore, research on complicated diseases often provides insights into other rare or common diseases, leading to other advancements.

Ninety percent of rare diseases still lack FDA-approved treatments, including our family’s disease of “choice,” PSC (primary sclerosing cholangitis). You likely know someone who suffers from a rare disease, whether or not they have revealed that to you. If you have no connection to a rare disease, celebrate your health and the knowledge that progress made here aids other conditions.

You can support Rare Disease Day by wearing zebra stripes (or fish stripes — I recently spied some tropical fish bearing zebra coloration while snorkeling in Hawaii). Use the social media hashtags #PSCPartners, #showyourstripes and #rarediseaseday. Or tag PSC Partners and NORD in your social media posts:

» Facebook: @PSCPartnersSeekingACure @rarediseases

» Twitter: @PSCPartners @RareDiseases

» Instagram: @psc.partners @rarediseasedayus

— Karen Telleen-Lawton serves seniors and pre-seniors as the principal of Decisive Path Fee-Only Financial Advisory in Santa Barbara. You can reach her with your financial planning questions at [email protected]. Click here to read previous columns. The opinions expressed are her own.

Support Noozhawk Today!

Our professional journalists work tirelessly to report on local news so you can be more informed and engaged in your community. This quality, local reporting is free for you to read and share, but it's not free to produce.

You count on us to deliver timely, relevant local news, 24/7. Can we count on you to invest in our newsroom and help secure its future?

We provide special member benefits to show how much we appreciate your support.

Email
I would like give...
Great! You're joining as a Red-Tailed Hawk!
  • Ask
  • Vote
  • Investigate
  • Answer

Noozhawk Asks: What’s Your Question?

Welcome to Noozhawk Asks, a new feature in which you ask the questions, you help decide what Noozhawk investigates, and you work with us to find the answers.

Here’s how it works: You share your questions with us in the nearby box. In some cases, we may work with you to find the answers. In others, we may ask you to vote on your top choices to help us narrow the scope. And we’ll be regularly asking you for your feedback on a specific issue or topic.

We also expect to work together with the reader who asked the winning questions to find the answer together. Noozhawk’s objective is to come at questions from a place of curiosity and openness, and we believe a transparent collaboration is the key to achieve it.

The results of our investigation will be published here in this Noozhawk Asks section. Once or twice a month, we plan to do a review of what was asked and answered.

Thanks for asking!

Click Here to Get Started >

Reader Comments

Noozhawk is no longer accepting reader comments on our articles. Click here for the announcement. Readers are instead invited to submit letters to the editor by emailing them to [email protected]. Please provide your full name and community, as well as contact information for verification purposes only.

Meet Your Realtor Sponsored by Village Properties

Photo of Dianne and Brianna Johnson
Dianne and Brianna Johnson
"We are committed to making our clients feel special by providing the concierge level service they deserve."

Full Profile >

Daily Noozhawk

Subscribe to Noozhawk's A.M. Report, our free e-Bulletin sent out every day at 4:15 a.m. with Noozhawk's top stories, hand-picked by the editors.

Sign Up Now >