debra of America Raises $600,000 at Annual Benefit; Honors Trailblazers in Epidermolysis Bullosa Community
10 Nov 2023
Drug ApprovalPhase 2Gene Therapy
DENVER, Nov. 10, 2023 /PRNewswire/ -- debra of America, the leading patient advocacy organization dedicated to improving the quality of life for those living with Epidermolysis Bullosa (EB), raised $600,000 at its annual benefit on November 4, 2023, in Atlanta at the Georgia Aquarium. Funds raised help advance EB research initiatives and directly support families and individuals living with EB.
"We are incredibly grateful to our donors and supporters who joined us on Saturday night to raise funds and greater awareness for EB," said Brett Kopelan, Executive Director of debra of America. "This disease is currently a lifelong sentence, and we truly believe we can move the needle to help more families and fund research that has the potential to improve quality of life and ultimately find a cure."
At the benefit, debra honored several EB trailblazers for their research and therapeutic contributions as well as individuals living with the disease. Award recipients included:
1st Annual Jouni Uitto Impact Award: M. Peter Marinkovich, MD
Spirit Award: Susan Craighead
Spirit Award: Analise Cardullo
Partners in Progress Award: BioMendics
Partners in Progress Award: Eliksa Therapeutics
Partners in Progress Award for First Approved Drug: Krystal Biotech
Dr. Marinkovich's contributions have made an indelible mark on EB therapeutic development including his role as the primary investigator for Krystal Biotech's GEM-3 Trial for the recently U.S. FDA-approved VYJUVEK™ for Dystrophic Epidermolysis Bullosa.
"Dr. Marinkovich is not only an exceptional clinician but also a pioneer in the field of gene therapy for treating EB at a molecular level. It is due to his groundbreaking work that we have seen the first FDA-approved treatment as well as other promising therapeutic approaches for this condition. Therefore, it is fitting that he receives the inaugural Jouni Uitto Impact Award," said Kopelan.
The Spirit Award, given to Craighead and Cardullo, recognizes individuals living with EB in the United States who possess great courage and strength in spite of the challenges this disease engenders. Craighead, born in 1964 with DEB inversa, defied the odds during a time when survival for infants with EB was extremely rare. Her outstanding academic achievements, unwavering commitment to community advocacy and distinguished career as a King County Superior Court judge in Washington state are a testament to her resilience and dedication. Analise Cardullo, diagnosed with Junctional Non-Herlitz EB from birth, has also shown unwavering determination in her life. She actively participates in varsity swimming, choir and farm work, refusing to let her condition define or slow her down physically. Her spirit and diverse pursuits showcase her remarkable resilience and determination.
Three Partners in Progress Awards were granted to BioMendics, Eliska Therapeutics and Krystal Biotech for their respective contributions to EB therapies and treatments. BioMendics is at the forefront of developing MTORX Technology, a topical gel tailored to target and eliminate mutated and damaged keratins in the skin of EB Simplex patients. Their upcoming TAMES Phase 2 clinical trial, slated to begin next spring, will encompass both children and adults with EBS. Eliska Therapeutics is currently pioneering the development of ELK-003, an innovative eye drop designed to address corneal abrasions in individuals with EB. ELK-003 holds the potential to revolutionize the treatment of ocular manifestations associated with EB. Eliska Therapeutics is determined in its commitment to advancing patient care and bringing this groundbreaking eye drop into clinical practice. Both of these companies have demonstrated an unwavering commitment to the community by actively engaging people living with EB to ensure their work will make a meaningful impact on their quality of life. Krystal Biotech was recognized for its work in developing VYJUVEK™ (beremagene geperpavec-svdt), the first FDA-approved treatment of Dystrophic Epidermolysis Bullosa (DEB). VYJUVEK is a topical gel that addresses the genetic cause of DEB by restoring functional copies of the COL7A1 gene to patients and is the only medicine available for patients in the U.S.
Following on the success of their benefit, Brett Kopelan will be a featured panelist in the RAAP 2023 Fall Policy Conference on Capitol Hill alongside policymakers and other foundation leaders to address access issues for those living with rare diseases. As part of its government and advocacy work, debra is frequently active on Capitol Hill, advocating for specific important pieces of legislation and to foster stronger relationships with federal government officials and relevant agencies to further its mission and impact.
Epidermolysis Bullosa is a rare genetic disorder characterized by fragile skin that easily blisters or forms painful sores from minor friction or trauma. It affects people equally across genders, ethnicities, races and all age groups and has significant physical, emotional and finanicial impacts on both patients and their families.
For more information about debra, visit www.debra.org.
About debra
debra of America (debra) is a nonprofit organization dedicated to enhancing the lives of individuals and families impacted by Epidermolysis Bullosa (EB), a rare genetic disorder. The organization integrates direct services, advocacy, education and research to foster meaningful change for EB patients. Epidermolysis Bullosa is characterized by fragile skin prone to painful blisters from minor friction. debra provides vital resources, builds community and drives research to alleviate the challenges associated with EB, supporting patients and their loved ones. With a longstanding commitment to EB awareness and study, debra is a pillar of support. By uniting caregivers, advocates and supporters, the organization envisions a future where the burdens of EB are lifted, empowering those affected to lead lives of greater comfort. For more information about debra, visit www.debra.org.
Media Contacts
Kelly Potts / Ryan Mudgett for debra of America
[email protected], (206) 343-1543
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