Global Genes Brings Hope To Victims of Rare Diseases

07/07/2016 04:33 pm ET Updated Jul 06, 2017
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What began as a labor of love for Global Genes CEO and founder Nicole Boice has grown into a global advocacy group that seeks to help over 350 million victims of rare disease worldwide. I had the opportunity to sit down with Nicole Boice in the wake of the announcement of the winners of RARE Patient Impact Grant Program, an inaugural event that directs $100,000 to deserving patient advocacy groups and rare disease foundations.

• What led you to be a champion for rare disease research and funding?

While I'm committed to the rare disease community, it's hard to call myself a champion. I am continuously humbled and inspired by rare patients and their families, how determined they are to find cures, fund research, and change the landscape. So, I think they are the real champions. And despite all their challenges and hardships they continue to persevere. I realize health is everything, and if we have our health, it is our responsibility to help those who don't. I came to this community when my dear friend's son was diagnosed with a rare disease. I witnessed her struggles, his struggles, and realized that they felt so alone on this journey. I started Global Genes in my garage as a grassroots, homegrown movement, with the purpose of trying to help connect patient advocates to one another, and give them the tools that they need to be successful on their journey. I'm amazed at how Global Genes has grown and evolved over the past years, but even with the impact that we have made so far, there is so much more that I know we can do. Patients and advocacy are at the heart of our efforts, and we are laser focused on ensuring that Global Genes continues to positively impact this community of millions in need. Failure is not an option.

• How does the grant program specifically impact patients and their families?

The RARE Patient Impact Grant program is designed to provide much needed funding for rare patient organizations and the programs that they are developing. They are best equipped to provide specialized resources, support, and education to their communities. This grant program provides Global Genes the ability to cast a wider net and allow patient organizations to help their communities. We provided $100,000 in grants to 18 different organizations in 2016. The projects will make a tangible difference in rare patient and caregiver's lives. For instance, no-cost oral replacement therapy for Moebius Syndrome families or adaptive cycling equipment provided through the Ataxian Athlete Initiative. Global Genes wants to do more. We received over $1.4 million in funding requests that were all worthy. Our goal is to continue to raise more money to meet the increasing needs, all in an effort to provide resources that increase a patient's quality of life.

• Describe the educational sessions, how do they further the awareness of rare diseases?

There are a number of educational programs funded through the grant that will create empowered and informed patients and caregivers. Global Genes believes knowledge is power and will prepare these families for their medical and advocacy journey. It is important to raise awareness about rare disease with accurate information and these educational sessions provide advocates an invaluable opportunity to connect with each other, share successful strategies for raising awareness and to access experts in the rare community.

• To potential donors - what types of programs will their donation service?

Global Genes has been very conscientious about the money that has been donated by individuals, $0.82 of every $1.00 directly supports programs that benefit the rare community. Some of the most highly utilized resources that Global Genes collaboratively produces include the RARE Toolkits and Webinars. Each year the topics for these materials are set by requests from the rare patient community. The signature event that provides advocates the full immersion experience is the RARE Patient Advocacy Summit. In 2015, this one-of-kind event brought together over 400 rare disease stakeholders representing 40 states and 6 countries and 120 unique rare diseases. Donations to Global Genes directly fund this programming in addition to the RARE Patient Impact Grant Program.

Groups such as NGLY1 Foundation, who advocate for the sufferers of Congenital Disorder of Glycosylation (CDG), were among the recipients for the RARE Patient Impact Grant Program. There are only 150 known cases of CDG in the United States, making advances in its treatment increasingly difficult to pursue. Thanks to Nicole Boice and Global Genes, research can now be funded for children who suffer from CDG's characteristic developmental delays, regular seizures, and liver dysfunction.

They are but a few of the research and advocacy groups who will benefit from this life changing grant program. Other awardees include: 1p36 Deletion Support & Awareness, ADCY5 Mutation, A Foundation Building Strength, Alternating Hemiplegia of Childhood, CADASIL Association, Children's Interstitial and Diffuse Lung Disease Foundation (chILD Foundation), Friedreich's Ataxia Research Alliance (FARA), Helping Hands for GAND, Inc., Histiocytosis Association, Inc., Hope for SCD, International FOXG1 Foundation, LHON Project at UMDF, Moebius Syndrome Foundation, National PKU News, Pachyonychia Congenita Project, and Usher Syndrome Coalition

Global Genes will accept grant applications for 2017 awards between July 15, 2016 and September 15, 2016. Types of projects funded include special programming conference support, disease specific educational resources for schools, adaptive equipment and technology for patients, and micro-grants for early investigative research.

Please visit https://globalgenes.org/give/ for more information on how to write a grant, give a donation or become involved in your community.