Spring 2010

Newsletter

Happy Spring!

Happy Spring! Welcome to our new newsletter format. We hope that your Winter was as healthy as could be. Please read below about our new Fundraising Challenge!! Sometimes it is easier to remain hopeful and positive if we are thinking positive thoughts and taking action.

Please note that many of our sections in the old newsletter are now listed as SDSF Important Links in this new format. For instance, if you want to find out the name of a Regional Parent Contact in your area, just click on the link to the right and you will see the list. I hope that this works out for everyone. It sure is more fun to put pictures into this format! I can't wait to upload Camp Sunshine pics! I am looking forward to seeing many of you at Camp Sunshine and for those of you who can't attend, we will be posting pictures and summary articles in the next newsletter.

Let us know what you think about this newsletter format by emailing our Administrative Assistant, Kim McDowell.
Sincerely,

Blair Van Brunt, President SDSF

CAMP SUNSHINE

Camp Sunshine is accepting
applications (click on link above for the application)on their web site for our SDS week this coming summer from July 25 - 30th. We have confirmation from 7 of 8 doctors/scientists who are able to come. These fabulous people are: Dr. Johanna Rommens, Dr. Blanche Alter, Dr. Richard Harris, Dr. Akiko Shimamura, Lynda Ellis, RN, and Dr. Elizabeth Kerr. We are awaiting word from Dr. Peter Durie. These wonderful doctors/scientists have been involved with SDS for a very long time and are not only tops in this field but are wonderful to talk with, ask questions of and learn from. We are so fortunate that they are able to take time from their busy schedules to come be with us for the first 2 days of the family meeting.These doctors represent some of the different fields that this syndrome presents in: hematology, gastro enterology (this is Dr. Peter Durie who we are awaiting word on), genetics, psychology, and bone marrow transplantation.
As it has been mentioned, Camp Sunshine handles all of the applications and the questions associated with them. We hope that you and your family will be able to join us this summer for time to SHARE, LAUGH & LEARN!!

In This Issue

Camp Sunshine

Fundraising Challenge

Fun-d Raising Ideas

NORD and the FDA

Participate in Research Studies

Planning a Fundraising Event

Florida Senate Bill

Thank you for your Donations

Article Headline

SDSF Important Links:

Donate to SDSF

Medical Scientific Advisory Board

Regional Parent Contacts

Doctor Database

Adult Patient Resources

Receive Hospital Care Basket

Please Join Patient Database

Participate in Research Studies

Purchase Your Own SDSF Bracelet

SDSF FUNDRAISING CHALLENGE

Do You Want A Cure?

Do You Want More Effective Medical Information and

Treatments For Your SDS Patient?

YES?

So do we at SDSF! We can help the dedicated doctors and scientists who are interested in doing the research on these kinds of projects by funding them. But we must all pitch in and send in money.

HAVE WE GOT A DEAL FOR YOU.......

We propose to host a fundraising contest and

the Winner will receive a Dell Laptop computer

We also have a Second prize of 2 Southwest Airlines tickets

redeemable anywhere in the continental United States

Here's the deal: Starting now (whenever you read this) through September 30^th, the person/family who raises the most money above and beyond $1,500 wins!

This money can come from corporations, family members, friends etc. All donations must be in check, cash, credit card through our web site (Paypal), and stock options, and may not be in-kind donations.

When you ask your family/friends/companies/churches/temples etc to send in their donations, please ask them to add a note that states who the donation is associated with ( this should be the name of the fundraising organizer) so that we can keep track of the funds that are sent in. The note can be on the check, in a separate email, separate piece of paper or a call to our 1-888-825-SDSF (7373) number. You can also ask them to send the donations to you and you can then send everything in at once with a note. Whatever works best for the donor. Just please do not create a seperate bank account for this purpose as it is against the law.

We are very excited about this contest and can't wait to see how our community will respond to not only helping their own family but helping all of us!

Please feel free to call 1-888-825-SDSF (7373) anytime to discuss questions, comments or possible fundraising ideas with us. I personally have done quite a bit of fundraising and look forward to helping you plan a strategy or event. You can also read the article below for some easy fundraising ideas. Remember, it does take money to make money but we can help you figure out a way to keep your costs down and how to recoup them.

Please refer to this link to review tips and thoughts on fundraising

please note that the offered prizes are acquired donations through Bryan Sample, Treasurer of SDSF.

MORE FUN FUNDRAISING IDEAS

SDS Rubber Bracelets can be bought through SDSF

and sold at any of these events below. We sell them for 10 @$25.00 and you can re-sell for whatever you feel your market will bear.

Alumni Reunions - sell SDSF Rubber or Crystal bracelets

Art Exhibit - of locals or kids - charge admission/sell tickets

Auction/Silent Auction -get donated items from friends, family and local businesses.

Bake Sale

Balloon Bouquets - buy wholesale, sell retail

Bazaar And Fair - charge for admission and food and drink

Block Party - sell rubber and crystal bracelets

Box Lunches -make them yourself and sell at retail

Breakfasts (Pancake,International Food, etc) - sell admissions or single meal

Car Wash - get volunteer students, ask fire dept for access to water

Celebrity Lunch Or Dinner -charge admission

Christmas Craft Sale

Christmas Wish Tree

Craft fair - charge admission and ask each vendor to donate a portion of their proceeds

College Final Exam Survival Kits -sell to friends/family

Community Festival Consignment Items Sale

Cookoff Contests (Dad& Daughter, etc.) - charge entry fee

Easter Gift Baskets for sale

Fashion Show Luncheon

Father/Daughter Dance -

Flower Sale -- Mother's Day

Game Contest Marathon

Guess Beans In Jar Count

Hobby Fair

Home Tours - charge admission

International Foods Sampling -charge admission

Letter Writing Campaign - perhaps the easiest of all! Click here to see sample letter that you can mail/email to family/friends and include our Donate page link in the email for fast and easy giving. Marathon or any kind of running event - check out separate article on this

Outdoor Concert

Poker Game- charge entry fee and re-supply of chips. Winner can donate winnings to SDSF

Pet Shows - charge admission and get donated prizes

Rummage/Garage sale

Supper With Ticket Sale (Chili, Hamburger,Spaghetti, Pizza, Barbecue, etc.)

Ticket Sales Of Door Prizes At Special Events

Tournaments/Events (golf, walk/run event,tennis, softball, chess, etc.)-see separate article

Used Book Sale

Wine And Cheese Tasting's - charge admission and donate portion of sales of wine to SDSF

Most of the events that need to charge admission/sell tickets can be priced by finding out what other charities charge for fundraising events in your area.

RARE DISEASE DAY EFFORTS ON BEHALF OF SDS

Rare Disease Day logo A PARENT AND A SCIENTIST

EACH MAKE A DIFFERENCE IN THEIR STATES SUPPORTING
RARE DISEASE DAY

The following is from Dr. Nikolaus Trede, a researcher in Utah who SDSF financially supported through a grant in 2008:

"My lab at the Huntsman Cancer Institute, University of Utah, works on a number of rare immunodeficiency diseases using the zebrafish model. These include Shwachman Diamond Syndrome (SDS), DiGeorge Syndrome and a variety of deficiencies of the innate and adaptive immune system that we have generated in the lab. In addition, I see children with rare oncologic and hematologic diseases in my clinical practice here at Primary Children's Hospital, University of Utah.

On February 2nd, I received the call to send a petition to my State Governor to declare February 28th Rare Disease Day from Blair Van Brunt, President of the Board of Directors of SDSF that supports our work on SDS. I felt that this was a wonderful idea to raise the awareness of the public for these diseases that, while uncommon, have important clinical and research implications and need a lot of support. I therefore sent the petition that same day to Utah Governor Gary Herbert's office. I was extremely pleased when I received a call from his office on March 1st informing me that the petition of three non-medical individuals and my own had caused Governor Herbert to enact February 28th Rare Disease Day in the State of Utah."

The following is from MaryAlice Savulich, parent of a child with SDS:

"As many of you may know February 28th has been proclaimed "Rare Disease Day" in our Nation. Recently, I learned that New Jersey had not yet joined the team to bring awareness to orphan and rare disease that sadly affects so many families on a national level. With little time to get the job done on February 2nd, I contacted New Jersey Governor, Chris Christie, in a letter as well as an email asking him to proclaim February 28th as "Rare Disease Day" in New Jersey. I am pleased to say that he responded and on February 17th, he proclaimed February 28th "Rare Disease Day" in New Jersey.

For me personally, it is a milestone. Our daughter became ill at the age of 5 weeks old and eventually was diagnosed with probable SDS. "Unusual" and "rare" were words frequently used when describing our daughters condition. It was frightening because in our case the word "rare" meant unknown. This is one of over 7,000 rare diseases, and although we have found support in our individual communities of diseases, we can now draw more awareness as a nation. My hope is that we can more easily share with one another our journeys and provide understanding to both those directly affected and their friends and families. The journey is not only an extremely emotional journey, but it can become a financial stuggle as well. Ultimately, knowledge reduces fear, and the the more opportunity to raise awareness of orphan disease the more the medical community can do to help our loved ones and the more our nation can reach out to help.

I want to say thank you to Blair Van Brunt for, unbeknownst to her, reminding me about February 28th, and thanks to her I was able to stop a minute and look a little closer to see how I could help our children and the children and adults of our future"

Thanks to both of these wonderful people and their efforts and to those who may have achieved something in their state as well.

NORD AND THE FDA NEWS

The following is an excerpt from the National Organization of Rare Disorders:

Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD), has announced that the Food and Drug Administration (FDA) has created a new position-Associate Director for Rare Diseases-in the agency's Center for Drug Evaluation and Research's (CDER) Office of New Drugs (OND) represents an important advance for patients with rare diseases.

"NORD asked FDA to create a new position within CDER to provide a focal point for the approval of new orphan drugs, and to supplement the support that orphan products receive from the Commissioner's office," Saltonstall said. "We believe the creation of this new office reaffirms and expands FDA's commitment to facilitate the development and approval of safe and effective drugs for Americans with rare diseases."

Dr. Anne Pariser has been selected as the new Acting Associate Director for Rare Diseases and the agency said it will move quickly to fill the position on a permanent basis. Dr. Pariser will report to the Director of the Office of New Drugs, Dr. John Jenkins.

The FDA said that the Associate Director for Rare Diseases will serve as CDER's "focal point to the rare disease drug development community and assist stakeholders and developers of drug and biologic products in navigating the complex regulatory requirements for bringing safe and effective treatments to patients in need."

The Associate Director will also coordinate an initiative to develop CDER policies and procedures for the review and approval of treatments for rare diseases. An important focus of this new initiative, according to the FDA, will be to ensure collaboration among scientists and clinicians throughout CDER to help develop and bring to market new treatments for patients with rare diseases.

CAN YOU SEND IN TISSUE? BLOOD? PATIENT INFORMATION?

SDSF has a long history of many families participating in different studies that researchers have proposed and are needing tissue, blood or information of an affected SDS patient. Click here to see what the current needs are

EXCITING NEWS FROM ONE OF OUR MEDICAL ADVISORY BOARD MEMBERS!!

Dr. Monica Bessler

Dr. Monica Bessler, a member of our 13-member Medical Advisory Board, has announced that she will be leaving St. Louis, MO for Pennsylvania! Here is an article by Dr. Bessler on her new adventure:

New Bone Marrow Failure Center Announced

The Children's Hospital of Philadelphia (CHOP) and Hospital of the University of Pennsylvania (UPENN) have recruited Dr. Monica Bessler MD PhD, and Dr. Philip J Mason PhD to initiate The Pediatric & Adult Comprehensive Bone Marrow Failure Center at CHOP and UPENN. In July 2010 Dr. Bessler will take up the position as the Director of this new center and program. "This is an exciting and unique opportunity for us to bring together brilliant scientists and outstanding physicians in Pediatric and Adult Hematology to jointly investigate the underlying mechanisms of bone marrow failure (BMF), improve its diagnosis, explore new forms of treatment and mentor a new generation of students and physicians in the front-line investigation and care of patients with BMF and their families."

An additional focus of the program will be to facilitate the transition from pediatric to adult patient care. "With the achievements in science and medicine and the improvement of patient care pediatric patients survive to be adults, in addition more adult patients are diagnosed with BMF because of newly discovered inherited predisposition genes. It is therefore important to provide more comprehensive lifetime care for patients with BMF and their families and to train "pediatric" and "adult" physicians in training in the care of pediatric and adult individuals with BMF ".

Dr. Bessler will further introduce with more details the CHOP/UPENN Comprehensive BMF Center in the SDS Fall News Letter. But we will get a chance to talk with her about this sooner as she is coming to Camp Sunshine this summer!!

INTERESTED IN PLANNING A GOLF/WALK/RUN/ BIKE EVENT?

Want to plan an event in 6 weeks? Here's a guide:

6 weeks out: Determine what event you think will work best for you and Ask for Help! Ask your family, friends and colleagues to volunteer to help. You'll want 2-3 people minimum as your organizing committee.
5 weeks out: Get Organized! Set a location, date & time. Determine a fundraising goal, and set up a fundraising web page (if you can) and create your invitation. Ask local businesses to sponsor/donate to your event. If you can't get dollars, get in-kind donations. Order SDS rubber bracelets or create and order t-shirts from your local supplier.
4 weeks out: Spread the Word! Email/mail your invitation. Ask friends and family to volunteer to help at the event. Reach out to civic & religious organizations you are involved with. Contact news media.
3 weeks out: Check Final Details! Print signs and other materials for event location. Gather donations of refreshments. Stay in touch with your volunteers.
2 weeks out: The Home Stretch! Send reminders to registrants and volunteers. Double check supplies and materials.
1 week out: Countdown to Success! Check weather reports. Verify contingency plans. Confirm volunteer assignments.

To get the word out and to help with registration, you could set up a blog for your race (Wordpress) which can be continually updated by you without having to have any web language and then use a Paypal button that links to our account for instant deposits for the registration fee.
www.firstgiving.com does help you set up a web page for an event and many non profits use this. There is another family who uses a web site www.active.com. to register people who want to run in their 5K race. They get corporate sponsors for their race as well: US Track & Field in their area, etc. Another web site to use is www.setupevents.com which will set up the registering, do the timing if you want and so much more. They are strictly a triathlon event company. www.signmeup.com is another web site but I do not know much about them. Worth investigating!

Of course, most of them have fees but it always takes money to make money and you can possibly recoup the costs by sending us an accounting of your expenditures so we can possibly reimburse you if you can't find a donor for that purpose!! Usually a family member, friend or company or someone can help:)

If you know someone who has a Constant Contact account, you could get them to create an email for you to announce the event and then you can use it to print posters and continue to change it a bit to send out new information about the event as it gets closer. You can also put our Paypal link in that email.

FLORIDA STATE SENATE AND HOUSE SET TO PASS BILL ABOUT PANCREATIC ENZYMES AND STUDENTS' ABILITY TO CARRY THEM BY THEMSELVES IN SCHOOL

Florida Senator Bill Wise introduced a Bill SB166 that would eliminate the need for students to go to the nurse's office for their pancreatic enzyme medications prior to a meal but would allow them to carry the enzymes with them in their bags/purses without fear of reciprocation by the school authorities. This bill needed and has now received the approval of the State Board of Education and has gone to the Senate Special Order Calendar to await the next step.

I am not sure what the next steps after this are but it does give all of us information on how to propose this to our own State Senators and Representatives for adoption in our own states. Here are a few sections of the bill. Please click on the link above to see the whole bill.

I. Summary:

The bill authorizes a student to carry and self-administer prescribed pancreatic enzyme supplements while in school, at a school-sponsored activity, or while in transit to or from school or a school-sponsored activity, if the student's parent and a prescribing practitioner have provided the school with authorization for the student's use of the supplement. The State Board of Education, in cooperation with the Department of Health, would be required to adopt rules for the use of the supplements. The bill requires the parents of a student who uses prescribed pancreatic enzyme supplements to indemnify the school district, county health department, public-private partners, and their employees or volunteers for any and all liabilities arising from the student's use of the supplements.

This bill amends section 1002.20, Florida Statutes. BILL: SB 166 Page 2 with cystic fibrosis.2 Shwachman-Diamond Syndrome is the second most common cause of inherited pancreatic insufficiency.

III. Effect of Proposed Changes:

Students with, or at risk of, pancreatic insufficiency or students with cystic fibrosis, who need the enzymes to grow and develop appropriately, would be authorized to carry and administer enzyme supplements at school or school-sponsored activities with parental and medical authorization. This authorization would permit the student to take the enzymes in a timely manner when they ate food containing fat, protein, or complex carbohydrates. The State Board of Education, in cooperation with the Department of Health, would adopt rules for the use of the supplements. The bill provides an affirmative right to carry and use the supplements. Since s. 1006.062, F.S., requires school districts to adopt policies governing the administration of medication on their campuses and at events, the requirement for the state board to adopt rules may be superfluous. Parents who grant the authorization would be required to indemnify the school district, county health department, public-private partners and their employees or volunteers for any liabilities arising from the use of the supplements. Indemnity clauses do not eliminate a party's liability; rather, they operate to allow the party to seek restitution from another. In a school setting, a parent's ability to indemnify the school district or another party may be quite limited.

The effective date of the bill is July 1, 2010.

THANK YOU FOR YOUR DONATION (November 19, 2009 - February 28, 2010)

IBM Employee Services Center

Nan & John Galbreath

Rose & Frank Canonico

Roy Hohn

In Honor of Gracie Van Brunt - Gracie Fund

In Honor of Christopher Garfield - Bryan & Stefanie Holloway

In Honor of Kaitlynn Bright - Lori Ess

In Honor of Kaitlynn Bright - Rita Sussko

In Honor of Collin Brown - Debra Salter

In Honor of Collin Brown - Pat Murdock