Rare Disease Awareness Network

Together We Can
AUGUST 17, 2012 VOLUME 5, NUMBER 1

Welcome!
Can you believe that we are already publishing our fifth edition of the Rare Disease Awareness Networks newsletter? We have come so far in such a short while!

In the News Five Reasons to Cheer

I will let you in on a little secret about my past that may or may not surprise youI was a cheerleader! I find it funny that Jenn DaCosta and Patti McArdle, members of our board, were also cheerleaders. Patti is still involved as a cheer coach even. And my sister Jen, who swore she would never cheer in her lifetime, is a cheer mom! So when we come across an article about Rare Diseases and it the title says we should cheer, well thats exactly what we do!! The following is a brief version of the Scientific American blog article written by Ricki Lewis. The progress being made on behalf of the Rare Disease community is definitely something to cheer about! Rare Diseases: 5 Recent Reasons to Cheer By Ricki Lewis | July 29, 2012| 1. Gene Therapy Pending Approval: the very first gene therapy program has gained the impending approval of the European Medicines Agency. Why is this significant? Well, prior to this, gene therapy had been denied by all governing bodies. That means that pharmaceutical companies would not pursue it as viable treatment options. The very first approval opens the door for more research and hopefully, cures. Finding Homes for Stalled Drugs: The idea is simple yet brilliant: match compounds that are languishing on company shelves to diseases with newlydiscovered mechanisms. Such candidate drugs have passed initial safety tests but were dropped for business reasons, such as a tiny market, or because they didnt treat what they were intended to. Speeding FDA Approval: On July 9, President Obama signed into law the FDA Safety and Innovation Act, which updates the 1983 Orphan Drug Act. The new law provides $6 billion over the next 5 years to assist the agency in evaluating new drugs and medical devices. The Act will speed access to new treatments and development of especially promising ones, and the Humanitarian Use Devices program will target those that treat rare diseases, giving priority to diseases of children. Easing Insurance Access: In June, the Affordable Care Act was signed. It means patients will not be penalized for their pre-existing conditions, nor face annual or lifetime insurance caps. Identifying Disease Genes: New exome-derived discoveries are being reported nearly weekly, some appearing in the media before the technical papers are published.

Our mission is to provide awareness, advocacy, and education regarding all aspects of Rare Diseases.

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Rare Diseases: 5 Recent Reasons to Cheer was published by Scientific American on July 29, 2012 and was written by Ricki Lewis. Please check out the entire blog on their website.

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Spotlight on Pancreatic Cancer

Written By Ronda Eagle

One of the first things people always say to me when they find out my mom, Sandy, had pancreatic cancer is I didnt know that pancreatic cancer is a rare disease. The honest truth is, neither did I. Pancreatic Cancer became very popularized when Patrick Swayze was diagnosed in March 2008. Later that same year, in a much less popularized fashion, my mother was diagnosed as well. My mother was always quite energetic and could run circles around people half her age (I speak here from experience). Looking back, we began to know something was wrong at the end of 2007 when I moved to South Carolina. My parents had come up to visit and help me settle in to my new surroundings. While they were here, I noticed she had a marked lack of energy, had lost a lot of weight and had no appetite. Shortly thereafter, we convinced her that she should see a doctor because something was just not right. My mom was diagnosed with pancreatic cancer in June 2008. Treatment for pancreatic cancer is often very limited. The vague symptoms make it difficult to diagnose in its early stages. In the later stages, the tumors are inoperable leaving chemotherapy or chemotherapy and radiation as the only viable treatment options. Pancreatic cancer is the 4th leading cause of cancer death because there is no approved early detection tool used to diagnose it in its earliest stages when tumor removal is still possible. That is until now. In May of this year, a 15 year old High School Student from Maryland, named Jack Andracka, won first place at the Intel International Science Fair for his invention of a simple dipstick blood and urine test which can detect pancreatic cancer during its earliest stages with 90% accuracy. It is also 28 times cheaper and 1000 times more sensitive than existing tests for pancreatic cancer. I feel Jacks invention should bring about feelings of hope and promise not only to the pancreatic cancer community but to the entire rare disease community. His invention says to me that there are answers out there somewhere just over our horizon. We just need brilliant young minds, such as Jack Andracka, to find them.

Ronda Eagle isnt the only member of our Facebook family who has been affected by Pancreatic Cancer. Paula Almeidas uncle is fighting Pancreatic Cancer right now. Donna Ridolfi and Chris Green also lost their grandfathers to this cancer. Our hearts go out to all those who have been touched by this disease. Here are a few facts about Pancreatic Cancer:
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44,000 people will be diagnosed this year 37,000 will die from this cancer this year Symptoms are vague and include weight loss, jaundice, nausea, and diabetes Treatment options include surgical removal of the tumor if caught early enough; chemotherapy; and radiation

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Hope and Promise

Hope and promiselove those words! Please join us in our continued quest and hope that in our immediate future more screenings and testing procedures will be developed and others will commit to the promise of a brighter future for all Rare Disease patients.

Something for the kids Herding Zebra

Looking for a fun game to play with the kids on a rainy day when you are stuck inside? How about you herd zebra? Heres everything you need to play! Materials: Zebra striped balloons (found at most party blown up Two brooms Two laundry baskets Kitchen timer set to three minutes stores),

Directions: Lay the laundry baskets on their sides at either end of the living room or hallway. Blow up an uneven number of balloons (such as 7) and place them in the middle of the room or hall. Hand a broom to each child and assign them a laundry basket. The children then sweep the balloons into the laundry baskets. Whoever herds the most zebra balloons into their basket at the end on three minutes is the winner!

Upcoming Events
August 15RDANs 5th Newsletter publication date August 31wear ZEBRA and show your support for Rare Disease Awareness September: Childhood Cancer Awareness Month; Atrial Fibrillation Awareness Month; Leukemia and Lymphoma Awareness Month; Ovarian and Gynecologic Cancer Awareness Month; Prostate Cancer Awareness Month; Sickle Cell Month; and Newborn Screening Awareness Month September 3Labor Day September 8-9Visit RDAN at Acushnets Apple Peach Festival to buy your Rare Disease Apparel and Tote Bags

RDAN Board: Rebecca Alves, President Susan Green, VP Jamie Alves, Secretary Jennifer DaCosta, Treasurer Rosemary Arruda, Director Dawn Barksdale RN, Director Jennifer Bolarinho, Director Liz Costa, Director Patricia McArdle Esq., Director

Contact Information:
Please feel free to contact us at any time. Send us an email at savebecka@comcast.net. Also, we can be reached through the mail at: Rare Disease Awareness Network PO Box 30061 Acushnet, Massachusetts 02743

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Thank You!!
We cannot thank you enough for your support!! We hope you have enjoyed a few moments with us and hope you will be back for more next month! Is there anything you would like to see in the September RDAN newsletter? Let us know! Thanks a bunch!!