The Cystinosis Foundation continues to blaze patient advocacy paths. creating awareness of the needs of cystinosis patients at home and around the world and organizing a unique educational conference program. Our executive director, Valerie Hotz, recently attended the 28th Annual Benefit Gala hosted by Cystinosis Foundation of New Jersey and the EURORDIS Black Pearl Gala in Brussels, Belgium.
CYSTINOSIS FOUNDATION NEW JERSEY - 28 YEARS RAISING FUNDS
Over the past 28 years our friends in New Jersey have worked industriously raising funds to support vital research leading to significant early advances in our understanding of cystinosis and treatments for this rare disease. We owe a debt of gratitude to the Cystinosis Foundation of New Jersey for their hard work and all our supporters who care so deeply and direct their philanthropic gift to our shared vital mission.
The Cystinosis Foundation is an Associate Member of EURORDIS and Valerie attended the Black Pearl Gala in Brussels, Belgium on Rare Disease Day, February 29. Valerie was invited to join Patrick Reichenberger, Vice President, Commercial Operations for Raptor Pharmaceutical Corp., and Henk Doude van Troostwijk, Manager of Raptor's European operations at their table. Also in attendance representing our community were Antje and Jens Sgundek of Germany, Laetitia Lepoix, president of Cystinose France.
The Cystinosis Foundation's presence supported government approval of long-acting cysteamine - RP 103 - and encourage insurers and health care systems to approve reimbursement for this life saving medication that dramatically improves compliance, resulting in an improved quality of life for cystinosis patients and their families.
EURORDIS honored our longtime industry partner, Sigma-Tau Pharmaceuticals. Receiving Orphan Drug Designation in 1984, in the United States Sigma-Tau was an early leader in developing medicines for rare diseases, including developing an eye collyrium to treat ocular manifestations of cystinosis. We anticipate FDA approval of Cystaran sometime this year.
Last year the Cystinosis Foundatioin partnered with EURORDIS and NORD to include Cystinosis in Rare Connect, an online social network of rare disease communities. We hope you will visit the site at: www.eurordis.org and join the Rare Connect community.
We continue working to improve the quality of life for cystinosis patients every day and encourage you to live in the present moment. As Laura Chichelo, Cystiniosis Foundation director and sister of Gerardina Lotrecchiano wrote, "Yesterday was a good day, today is moving along nicely and tomorrow is anyone's guess. Living each moment to the fullest is our only guarantee.**
Kind regards,
Jean Hobbs-Hotz
President
More information can be found on our website:
Cystinosis Foundation, Inc.
58 Miramonte Dr.
Moraga, CA 94556
USA
The Cystinosis Foundation is a 501 (C)(3) nonprofit charitable organization. Our federal tax ID is 94-2927892. |
