Saturday, February 28, 2009

Today is Rare Disease Day

rare disease day

The last day of February has been designated as World “Rare Disease Day” to call attention to the public health issues associated with rare diseases. The Project Charity — The Children’s Rare Disease Network has compiled some facts and figures about rare disease that we thought would be of interest. If you have other facts and figures not on our list, please send them to us. We are particularly interested in international facts on rare disease that do not seem to be available.

Please visit the Official Gallery page to see victims of rare disease.


* Approximately 7,000 rare disorders are known to exist and new ones are discovered each year

* Rare disease affects between 25-30 million people in the United States and approximately 30 million people in the European Union

* One in 10 Americans is living with a rare disease

* Children represent the vast majority of those afflicted with rare disease

* Approximately 80 percent of rare diseases are not acquired; they are inherited. They are caused by mutations or defects in genes

* In the United States, rare diseases are defined as those affecting 200,000 or fewer people or about 1 per 1,000

* Rare disease is often referred to as an “orphan” disease

* Orphan or rare diseases are often not pursued by the pharmaceutical industry because they provide little financial incentive for the private sector to make and market new medications to treat or prevent them and because there are not enough patients to make research cost-effective

* Research on rare diseases can often lead to advances in our understanding of common diseases such as cancer, heart disease, diabetes, stroke and other major health problems

* As a whole, rare diseases represent a large medical challenge. Combine this with the lack of financial incentives to treat or cure rare diseases, and a serious public health issue is created

* The US Orphan Drug Act (ODA) of 1983 has been one of the most successful pieces of health related legislation ever enacted in the United States. Through a system of tax credits, government grants, assistance for clinical research, as well as seven years marketing exclusivity, the Orphan Drug Act has resulted in hundreds of approved orphan medicines, treating over millions of patients worldwide. Similar legislation has been adopted in Japan, Australia and the UK

* The US Orphan Drug Act has also been a detriment to healthcare. Many drug companies have accepted the challenge to be primary providers of rare disease drugs. Because they have no regulation and control over cost of medicines or competition (they are allowed to sell it without competition for 7 years), they can charge astronomical amounts of money for yearly treatments resulting in an extremly profitable business. Insurance companies often do not help pay for many of these drugs for victims of rare diseases who are dependant on the drugs for survival.

So, as we can see, rare disease are not that rare and need just as much support as well known diseases, like Breast Cancer.

Take a small moment to to pray for victims and research advancements. Make a small donation, put names on a prayer roll or write words of encouragement to a family who is struggling. Here are a couple of children that could use some love:

Hannah was born in July 2008. After birth, she had an enlarged spleen, very low platelets, and an enlarged liver. After many months of tests to try and find a diagnosis, a skin biopsy has diagnosed her with Gaucher's Disease, Type 2 or 3, a very rare genetic metabolic lysosomal storage disease with neurological and physical involvement and a life-expectancy of 2 (type 2) 15 to 20 (type 3) years or so. Hannah's mother is trying to spread the word about Gaucher's Type 2/3 Disease and any support you can give would be much appreciated. Visit her blog to find out about her story.

baby mia heart transplant Critical Aortic Stenosis

Baby Mia was diagnosed while still in her mother's womb with a rare heart disease called Critical Aortic Stenosis. She has had multiple surgeries, a heart transplant and many other complications. The McDonalds have insurance but after heart operations, a heart transplant, plus all the doctor visits and a lifetime of medicine... it would financially destroy the best of us, so any donations would be so helpful (they also accept giveaway donations to help raise funds). Visit HERE to find out about this special family and how you can help.

baby cora paige playground fundraiser

Cora Paige was a beautiful 10 month old girl that shortly passed away after being diagnosed with stage 4 cancer for only 3 weeks. Her parents are working to build a playground in her honor, to leave a legacy that will spread joy to other children. 100% of the proceeds of the sale of my Sugar Garland Necklace (shown below) will go directly to that fund, many other Etsy sellers have similar donations, search "Cora Paige" to find them. Read about it HERE. So far, $15,667.93 has been raised for this fund.

colorful vintage retro inspired kitsch multi layered  flower necklace

I know that there are SOOO many others out there, but these are the few I know of. Please take a moment to embrace your loved ones right now and enjoy them today in appreciation for the blessings we have in our families.


Robert said...

How nice of you to dedicate time to a post like this. It's always good to increase the awareness of such things!

Meekiyu said...

That's awesome post. Being in the medical field it does make one sad to learn of the many disease that people are unaware of that have devastating effects. Kudos to you on bringing this up.

Aisha said...

Such a wonderful yet heartwrenching post. Even though these stories make you sad, we should still be aware. Thanks for doing that, Lisa! Cora's story especially broke my heart. I pray that all the other little boys and girls and their families get through their tough days.

The Empty Envelope said...

Very sad, but much needed post! Those babies are so beautiful.

elsiee said...

Thank you for brining attention to these terrible illness that whose research is underfunded or non-existent because they are considered rare - of course they are an all to painful reality to the kids family.

so glad I discovered your blog on the ETSY forum thread! come visit me when you have a chance:

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