GFPD Fall 2013 E-Newsletter


GFPD NEWS IN BRIEF
  • If you’ve visited us online lately, you may have noticed our new look. We rolled out our new website this summer in order to reflect a greater emphasis on our community of families and children affected by PBD. You’ll find new Family Stories, a Kids Gallery, updated Blog and Resources pages, and links to all of our communities online. Take a look: http://www.thegfpd.org.
  • Did you #PauseforPBD on October 5th? We were so delighted to be joined by so many friends of the GFPD as we celebrated our fifth anniversary by taking a moment to pause and reflect on all we’ve accomplished and all the work left to do. Supporters posted and tweeted photos and messages of support as they paused and lit candles, sang songs of remembrance, and spread awareness in honor of their loved ones.  In addition to this day of reflection, we invited people to support the GFPD through a t-shirt fundraiser. By popular request, we’ve relaunched the campaign, but time is limited. Visit http://www.bonfirefunds.com/pauseforpbd to purchase your shirts by Wednesday, December 2nd!   
  • If you are shopping on Amazon this holiday season, please start at smile.amazon.com and then select "Global Foundation for Peroxisomal Disorders" as your designated charity. It will then direct you back to Amazon to start shopping and .5% of your purchase total will go to the GFPD at no cost to you! All the features you're used to, like Amazon Prime and Wish List, still apply. This is an easy way to support the GFPD!
 
GFPD CONFERENCES FOR FAMILIES AND SCIENTISTS

Over the summer, the GFPD hosted its fourth Family and Scientific Conference, bringing together families affected by peroxisomal disorders as well as researchers and clinicians invested in developing treatments for patients. Participants from around the world gathered in Omaha, NE for presentations by leaders in the field, as well as for family oriented discussions about topics relating to education, health management, and grief issues. Given the rarity of PBD-ZSD, families are typically isolated from others impacted by the disorder, and the GFPD conference is one exceptional opportunity for families to get together and bond over shared experiences. Likewise, researchers working on peroxisomal disorders are generally isolated from the population they seek to serve. With this gathering, researchers and families had the opportunity to interact and further each others’ understanding of various aspects of the disorder. And it’s not just in the interest of advancing the scientific understanding of the disease that brings scientists and physicians to our meetings; many members of the professional community come year after year to our events and have developed personal relationships with our children with PBD-ZSD, as well as their parents and siblings.  
 
The GFPD was grateful to receive a grant from biopharmaceutical company Retrophin to help offset costs to families who wanted to attend. Additionally, the GFPD provided family scholarships to ensure no family who wished to participate would be excluded for financial reasons. Fundraising efforts held throughout the year by the GFPD itself and by our families help to support these conference attendee scholarships.
 
This year our families were also joined by attendees of the International Peroxisome and Leukodystrophy Meeting, which was co-hosted by the GFPD and United Leukodystrophy Foundation in conjunction with our individual family conferences. Attendees came from Europe, Asia, and North America to give presentations on their work and collaborate with colleagues. Many stayed beyond the end of their meetings in order to get to know our children with PBD-ZSD and learn more about them from their parents, and to educate families about the work they're pursuing in their labs.
 
To see a photo gallery from the event, visit:
 http://www.thegfpd.org/ - !events-gallery-2015-conference/c1xpk
 
To see the meeting agendas or to view presentation slides and videos, visit:
http://www.thegfpd.org/#!2015-family--scientific-conference/cchv
http://www.thegfpd.org/#!2015-ipl-meeting/c1yt8
 
 
GFPD RESEARCH UPDATE
 
In a years-long effort, GFPD Family and Scientific Liaison Dr. Mousumi Bose has headed up a collaborative project with the GFPD Medical and Scientific Advisory Board, to prepare treatment guidelines for the PBD-ZSD population. Dr. Bose explains, "The expertise and insight documented in these guidelines will be a tremendous resource, as local physicians and specialists will, for the first time, have access to best practices and information about treatment and patient outcomes.” The manuscript, “Peroxisome Biogenesis Disorders in the Zellweger Spectrum: an overview of current diagnosis, clinical manifestations, and treatment guidelines” has been accepted for publication by Molecular Genetics and Metabolism. We are happy to extend our congratulations to the authors of this manuscript and look forward to announcing its publication soon.
 
A recent article published by the journal Stem Cell Research & Therapy  <http://www.stemcellres.com/content/6/1/158> describes research made possible in part by a GFPD grant awarded in 2013 to Dr. Joseph Hacia of University of Southern California to investigate neural cell resources for PBD-ZSD drug testing. This $50,000 award helped allow Dr. Hacia's research group to generate induced pluripotent stem cells (iPSCs) from skin fibroblast samples of patients with PBDs in order to develop more relevant drug testing and screening models for candidate therapies. In this paper, Dr. Hacia's research group used these iPSCs to generate patient-specific neural and liver cells, which are overwhelmingly affected in PBD patients. They demonstrated that these skin-derived neural and liver cells had peroxisome assembly defects and biochemical abnormalities that are typically found in PBD patients. This discovery opens the door for new models for drug testing and screening, including the high throughput screening that Dr. Hacia is already undertaking in his laboratory in collaboration with Dr. Jim Inglese at the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH) in Rockville, Maryland and Dr. Nancy Braverman at McGill University in Montreal. Dr. Hacia and his collaborators have expressed their deep appreciation for the generous support of the GFPD and are dedicated to finding better treatments for PBD-ZSD.


FAMILY AND COMMUNITY NEWS
 
We have been amazed and honored by the fundraising efforts we’ve seen from our families and friends of the GFPD in recent months. Our organization is funded entirely through the generous support of those who have been moved by their experiences with their own children or with other loved ones affected by PBD-ZSD and have sought ways to help. In one such recent effort, a work family at Schumacher Group—a healthcare staffing and hospital solutions company located in Lafayette, Louisiana—rallied around one of their own to show support in the face of unimaginable loss.
 
In early July, the company held a series of events to support Brant Gamble, Vice President of Business Development, and his wife Melissa, who is cofounder of the GFPD. Brant and Melissa lost their six-year old daughter Ginny to PBS-ZSD in April of this year, and since then, the couple has continued to celebrate Ginny’s life through continued fundraising efforts.  To join in that celebration, Schumacher Group organized a company-wide campaign to raise funds for the GFPD, starting with a butterfly release and a memorial walk in Ginny’s honor. Teams of co-workers dressed in pink for Ginny and gathered together to honor Ginny and all PBD-ZSD families. “We were so touched by the outpouring of support from Schumacher Group employees as we all came together to remember Ginny,” said Brant. “I am humbled to be part of an organization that truly supports their own.”   




 
Like Brant and Melissa, we too are so moved by the care and kindness demonstrated by members of the Schumacher Group family. We’d like to extend our thanks to the wonderful staff at Schumacher Group for their generous and thoughtful support and for their donation of over $5,000 raised through their efforts. You can read more about the Schumacher Group’s efforts to help remember Ginny and raise funds for the GFPD by visiting:
http://www.thegfpd.org/#!Schumacher-Group-Comes-Together-to-Celebrate-Ginny-Gamble/c1m5p/5653668a0cf29e70f223efd4.
 
Another family recently celebrated the lives of their children with a fundraising event. In Modesto, CA, friends of the Alfaro family came together to eat, drink, and be merry as they remembered the lives of Diego and Adrian, who were both affected by PBD-ZSD. Big brother Jose Jr. was excited to be the emcee this year and shared a few words about his brothers Diego and Adrian that enlightened the sold-out audience of 350 guests.




 
This year’s honored guest was Miss Ezra Burdick, who made the trip from Arizona to celebrate Diego and Adrian and help represent all children affected with PBD-ZSD. Ezra started the dance off with her father Jeremy in the “PBD Father-Daughter Dance.” Ezra’s friend and returning honored guest, Ainsley Wade, and her dad Kato also joined the dance floor and made the evening extra special.




 
The Dinner Dance featured live music by the Mike Torres Band as well as a raffle and silent auction that brought the evening’s fundraising total to over $22,000.  The dessert and silent auctions alone raised over $2,500 of that total, and was stocked with items donated by friends of the Alfaros and other members of the community. The Alfaros were thrilled with the support they received, noting that they “could not have had such a success without the help of our friends who volunteered or donated to the cause.” The Alfaros were also extremely excited to have GFPD Scientific Advisory Board member Dr. Joseph Hacia present, along with numerous PBD families—the Burdick, Wade, Haire, Gamble and Whitney families—who traveled from four states to attend.
 


To see more pictures from the Alfaro’s Dinner Dance, visit:
http://www.thegfpd.org/#!Family-Dinner-Dance-Fundraiser-Grows-in-its-Third-Year/c1m5p/565c98060cf2099ad66d3bcd
 
 
VIRTUAL FUNDRAISING LEVERAGES SOCIAL MEDIA
 
Not all fundraising was done in person this year. From the virtual Ilan-a-thon Jive K, which raised more than $3,000, to numerous Causes Wishes created by families celebrating their children's birthdays or other events, we've seen an uptick in fundraising through social media. One hugely successful effort came from the Chapman family of Illinois, who wanted to commemorate their son’s birthday by raising awareness about his disorder. In order to spread information about PBD-ZSD, Todd and Corin Chapman created a short video about Max and asked their friends to share it. With over 4,000 views on YouTube (and counting!) the video has already raised an astounding $20,000 to be used by the GFPD to fund research. Following a conversation with members of the research community at Family and Scientific Conference over the summer, Corin “left the conference with the intent of helping the GFPD raise $1 million,” needed “to advance any treatments currently in production.”
 
The Chapman family understands the importance of awareness. “Our PBD kids have a heartbreaking and inspiring story that rivals any television drama,” said Corin, when asked what drove her to create the video, “and I firmly believe the more we share it, the more funding and support we will receive.”

Watch the video and read more about how Todd and Corin made it at:
http://www.thegfpd.org/#!Family-Fundraising-Video-Raises-Big-Money-for-GFPD/c1m5p/56537e7f0cf29e70f2242335
 
 
CALL TO ACTION
 
Looking to get involved with the work of the GFPD? We’re looking for you! The GFPD is a volunteer-run organization, and we need your help to continue raising money for research, providing families with support, and spreading awareness about peroxisomal disorders and the mission of the GFPD.
 
Contact Communications Coordinator Heidi Harris (heidi@thegfpd.org) or Treasurer Melissa Gamble (melissa@thegfpd.org) for more information or to tell us how you’d like to get involved.
 

The GFPD family mourns the recent loss of the following children due to the devastating effects of PBD-ZSD:
 
Nina Serafini, November 19, 2013 — May 21, 2015 (Argentina)
Olivia Frank, July 31, 2006 — May 23, 2014 (West Virginia, USA)
Nord Iversen, January 15, 2014 — June 1, 2014 (Denmark)
Weston Klinger, December 19,
2014 — June 4, 2014 (Canada)
Leon Parker, July 8, 2013 — July 5, 2014 (United Kingdom)
Evangelia Malakis, February 27, 2014 — July 21, 2014 (Greece)
Harleigh Webster, March 21, 2014 — August 26, 2014 (Arkansas, USA)
Isabella Fitzpatrick, August 8, 2014 — September 30, 2014 (Ireland)
Summer Harrison, March7, 2013 — November 6, 2014 (United Kingdom)
Austin Lynch, June 22, 2012 — November 8, 2014 (Tennessee, USA)
Ghita Saghiri, November 3, 2014 — January 3, 2015 (Morocco)
Elianna Schultz, October 1, 2014 — January 8, 2015 (Canada)
Zaynab Elreda, October 7, 2014 — January 26, 2015 (Australia)
Ian Harris, March 18, 1993 — March 18, 2015 (Washington, USA)
Mason McGirr, December 16, 2012 — March 18, 2015 (United Kingdom)
Olivia Menard, February 9, 2011 — April 14, 2015 (North Carolina, USA)
Ginny Gamble, August 5, 2008 — April 25, 2015 (Oklahoma, USA)
Andrew Patrick, March 26, 2002 — April 29, 2015 (Ohio, USA)
Brayden Smith, September 6, 2014 — May 20, 2015 (Alabama, USA)
Killian Hall, June 13, 2011 — May 26, 2015 (Mississippi, USA)
Levi Mayhew, June 30, 2008 — July 24, 2015 (Indiana, USA)
Benjamin Edgar, April 8, 2002 —August 14, 2015 (Canada)
Luella Hale, October 3, 2014 — August 22, 2015 (Washington, USA)
Brittany Parrott, May 2, 1995 — October 30, 2015 (South Africa)
Willow Streak, November 1, 2015 — November 1, 2015 (United Kingdom)
Josh Boyd, December 14, 1995 — November 6, 2015 (Iowa, USA)
Radu Ianculescu, June 2, 2015 — November 9, 2015 (Romania)

 
Board of Directors
 
President
Shannon Butalla
shannon@thegfpd.org
 
Vice President
Anne Park Hopkins
annepark@thegfpd.org
 
Treasurer
Melissa Bryce Gamble
melissa@thegfpd.org
 
Secretary
Geoff Edgar
geoff@thegfpd.org
 
Medical/Scientific Liaison
Mousumi Bose
mousumi@thegfpd.org
 
Registry & Support Group Coordinator
Pamela Marshall
registry@thegfpd.org
 
Communications Coordinator
Heidi Harris
heidi@thegfpd.org
 
Woody Woodbury
woody@thegfpd.org
The GFPD is grateful for the input and guidance of our Medical and Scientific Advisory Board. For a complete list of members, please visit http://www.thegfpd.org/#!scientific-advisory-board/c1l1x.

Upcoming Events


Tee It Up for GFPD
Normal, IL
May 19, 2016

http://www.teeitup4gfpd.com
Contact: teeitup4gfpd@gmail.com

Ilan-a-thon
Baltimore, MD
June 4, 2016
ilanathon.org
Contact: Mousumi Bose: mousumi@thegfpd.org



Plan a Fundraising Event in Your Community

Please contact Shannon Butalla at shannon@thegfpd.org for more info.
Stay Connected

We have several active support communities online:


GFPD Parent Support (Parents & Guardians only)
PBD Dads: A Band of Brothers (Dads only)
GFPD Shutterfly Site (Parents & Guardians only)
GFPD Extended Family Support
GFPD “Public” Facebook Page
GFPD website
 
If your contact information has changed, please email Pamela Marshall at registry@thegfpd.org so we can update our records.

SUPPORT GFPD


GFPD is a 501 (c)(3) tax exempt public charity incorporated in the state of Oklahoma. By investing in GFPD, you are supporting children and families facing Peroxisomal Biogenesis Disorder-Zellweger Spectrum Disorder (PBD-ZSD) and assisting family members and professionals through educational programs, research, and support services.
 
All contributions to GFPD are tax-deductible (USA) in the year paid. Donations are accepted by mail or online at www.thegfpd.org.
 
Please mail checks to:
Global Foundation for Peroxisomal Disorders
5147 South Harvard Avenue, Suite 181
Tulsa, OK 74135





Designate contributions to GFPD through your employer's United Way Campaign or through the Combined Federal Campaign for Federal employees.  Contact Melissa Gamble melissa@thegfpd.org for more information.

Raise money for the GFPD when you search and shop online!

   
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