Vote4Hope Campaign – from Global Genes Project and Children’s Rare Disease Network

Pepsi Refresh Grant Competition inspires rare disease groups to compete for much needed funds!

Over the past few months we have had the good fortune of supporting a variety of rare disease initiatives that were all vying for Pepsi Refresh Grant monies.  The winners are chosen based on votes, and our rare disease community has done an amazing job being supportive of one another!

We in fact have made a concerted effort each month to promote those initiatives on rare disease tickets to ensure that every Pepsi Refresh competitor gets the attention it deserves.  It has been fun and inspiring to see the community work together!!

Now it is our turn -VOTE4HOPE.org!

Over the past two days – Vote4Hope  launched as a voting campaign centered around the Global Genes/CRDN submission for a Pepsi Refresh $250K Grant.   Global Genes/CRDN is developing The Global Genes Fund in conjunction with over 25 rare disease advocacy groups and parent advocates.  The Global Genes Fund is a technology platform that will exist to 1. Promote innovative ‘in their lifetime’ research and therapy development for kids affected by rare disease, to the general public 2. Create greater awareness about the prevalence of rare disease and the needs of the community at large.

This platform is being funded and developed by various underwriters including CRDN, the $250K grant win would help fast-forward the development of this new Fund, and ultimately be a win for the rare disease community as a whole.   Qualifying research will be included in this platform at no charge to the organization, providing an opportunity for rare disease organizations to garner new money and create new champions.   The Global Genes Fund is being developed ‘by the rare disease community  for the rare disease community’, this means we need the same energy and momentum behind it as the other disease specific rare disease initiatives.

Watch this video spot

http://www.crdnetwork.org/stories/videos/vote4hope_lg.html     Various videos have been crafted showing some of our kids taking ownership of this campaign as ‘their project’.  We are encouraging all patient and advocacy groups to do the same.  Join the campaign, get out the vote and make a huge difference in just 30 days – imagine. . .

Contact Nicole Boice with any additional questions regarding the Global Genes Fund at nicoleb@rareproject.org

The Global Genes Project Aims to Battle Rare Disease Through the ‘Vote4Hope’ Campaign and Pepsi Refresh Competition

The Global Genes Project Aims to Battle Rare Disease Through The ‘Vote4Hope’ Campaign and Pepsi Refresh Competition

Over 15 Million Children in America Are Estimated To Suffer From 7000 Unique Rare Diseases; Children Unite As ‘Ambassadors of Hope’ To Win $250K Pepsi Grant For The Global Genes Fund

DANA POINT, CA – September 1, 2010 – All Kids Deserve To Have Hope For A Cure – The ‘Vote4Hope’ Pepsi Refresh Rare Disease Campaign starts today. The Children’s Rare Disease Network (www.crdnetwork.org) and The Global Genes Project (www.globalgenesproject.org) are inviting the public, as well as caregivers, family and friends of the millions of children who suffer from rare diseases and disorders, to ‘Vote4Hope’ throughout September during The Pepsi Refresh Project.

The ‘Vote4Hope’ Rare Disease campaign is designed to help drive awareness for the unmet medical needs of the global rare disease community, and to support the development of the Global Genes Project. The Pepsi Refresh ‘Vote4Hope’ campaign opens today at http://www.refresheverything.com/fundhopeforsickkids and will continue through September 30.

“We entered The Pepsi Refresh grant competition with the goal of continuing education surrounding rare diseases and to generate the global awareness the rare disease community deserves,” said Nicole Boice, founder and president, Children’s Rare Disease Network and the Global Genes Project. “Many rare diseases affect small numbers of patients and because of the rarity of each condition, the public is often unaware of these chronic and life threatening conditions. However, collectively millions of people worldwide have rare diseases and everyone knows an adult or child suffering from a rare condition.”

If the Global Genes Project wins The Pepsi Refresh grant competition, funding will go to support further development of the Global Genes Fund. The Global Genes Fund is an innovative platform, which promotes as number of rare disease initiatives including ‘in-a child’s-lifetime’ research and therapy development. The development of the Global Genes Fund will provide tremendous value to the millions of children, families and rare disease organizations that support them.

Time To Refresh Rare Disease Facts

A staggering 250 million people worldwide are estimated to suffer from approximately 7,000 different forms of rare diseases with the vast majority having no therapies at all. In the United States, FDA statistics show that approximately 350 new drugs have been developed for the entire rare disease patient population since the passing of the Orphan Drug Act in 1983 despite incentives put in place by the federal government.

According to the National Institutes of Health (NIH), 30 million Americans are afflicted with a rare disease, or 1 in 10 people. The National Organization for Rare Disorders (NORD) estimates that of the 30 million people in the United States suffering from rare diseases, 50 percent or approximately 15 million, are children. Many rare diseases appear early in life, and about 30 percent of children with rare diseases will die before reaching their fifth birthday.

Rare Kids Become ‘Ambassadors of Hope’

Throughout the month of September and during the “Vote4Hope” Pepsi Refresh Rare Disease Campaign, pictures, stories and videos of children living with rare diseases will be featured on a daily basis on the Vote4Hope website: http://www.vote4hope.org/.

Called the ‘Ambassadors of Hope’, these children are representing the 7000 different rare conditions and suffer from diseases including: Ataxia-Telangiectasia, Autosomal Recessive Polycystic Kidney disease, Batten disease, Branchiootorenal Syndrome, Canavan disease, Cystic Fibrosis, Dravet Syndrome, Duchenne Muscular Dystrophy, Fibrous Dysplasia, Joubert Syndrome, Mitochondrial Encephalomyopathy, Niemann Pick Type C, Hutchinson–Gilford Progeria, Spinal Muscular Atrophy, Subcortical Band Heterotopia, Tay-Sachs and Type 2 Gaucher’s disease. To submit a child with a rare disease to the Ambassadors of Hope program, please visit the Vote4Hope website at http://www.vote4hope.org/.

“The ongoing lack of treatments for thousands of rare diseases has created an immense burden on the U.S. healthcare system and leaves millions of children and their families in despair,” added Boice. “At the Global Genes Project, our focus is on creating a unified voice and a platform for hope for this community, with continued efforts on educating the public and our government leaders on the importance of creating a national strategic plan for rare disease.”

About The Global Genes Project

The Global Genes Project is a campaign of the Children’s Rare Disease Network and is a grassroots effort with the goal to increase awareness about the prevalence and lack of treatments for rare diseases worldwide. The Children’s Rare Disease Network is a registered 501c3 non-profit organization. For more information, visit http://www.crdnetwork.org/ or http://www.globalgenesproject.org.

About The Pepsi Refresh Project

The Pepsi Refresh Project is giving America the power to decide how to fund good ideas, big and small, that help refresh our world. In an effort to support those who generate innovative, optimistic ideas, the Pepsi Refresh Project http://www.refresheverything.com, will award more than $20 million in 2010 to move communities forward. Each month, The Pepsi Refresh Project accepts 1,000 ideas and asks America to choose which ideas will win a Pepsi Refresh Grant.

For more information contact:
Jacqueline Tanzella
Spark Public Relations
jacqueline@sparkpr.com
415-321-1889

Nicole Boice
Global Genes Project
nicoleb@rareproject.org
949.248.RARE (7273)

Thank you Frances Perraudin – YES, It Is Time The World Pays Attention To Rare Disease!

Time Magazine and Frances Perraudin’s article, “Is It Time We Paid More Attention to Rare Diseases?” articulates the inherent challenges faced by the rare disease community and briefly introduces you to Carrie Ostrea and her daughter Hannah who suffers from a rare disease called Neuronopathic Gaucher’s disease.

Yes, a global rare disease registry is critical and Yes, pharma needs to recognize the importance of the rare disease market.

However, at the end of the day, the only way the rare disease community can win and we can help families like the Ostrea’s is to rally behind rare disease as one community of 250 million worldwide.  Many patient groups have seen the importance of  joining together, and there are new initiatives that are addressing the ‘awareness’ problem.

Unfortunately nothing moves fast enough for the rare disease community of parents trying to treat chronically ill and medically fragile children. We need to turn on the jets and start working to drive the needs of the rare disease community out to the masses.

The Global Genes Project, a collaborative and grassroots program working to raise greater awareness for rare disease, is educating the general public and building a platform to help fund research.

Thank you Time Magazine and Frances Perraudin for bringing the important subject of rare disease to the forefront and for your continued support.  We need ongoing media coverage to ensure that rare disease receives the global attention it truly deserves.

Nicole Boice

The Global Genes Project – Look Where We Have Come!

In January 2010, a collaborative project ensued called the Global Genes Project. The mission – to build a platform that would create greater awareness about the prevalence of rare disease and it’s impact on children and their families. Provide an educational platform to better inform those within the rare disease community and educate the greater public about disease, and lastly provide an umbrella platform for individual disease fundraising – creating a fun, innovative platform to help inspire new champions to help our community.

The team, comprised of about 8 parent advocates representing various diseases, including the amazing creative team from Charleston|Orwig, launched the initiative in January 2010, just in time to help promote World Rare Disease Day! In 4 weeks, over 150 organizations joined the effort, and the brilliant slogan, ‘hope. it’s in your genes’ was translated into 29 languages. Additionally, various disease organizations were able to utilize customizable resources to help aid in mini-fundraising efforts in their local areas.

What was so inspiring was the feedback from the community, and the proof that when we all come together, we can move mountains.

We have had additional organizations get involved and some join the Project committee! There are a lot of exciting announcements that will be made over the next couple of weeks – all of which will provide big opportunities for individual disease groups and to help continue to build traction within the general public.

Let’s keep this train rollin’! Send us feedback, thoughts, ideas, your successes and be part of the evolution and build out of the ‘next big cause’!

The Global Genes Team!

The Global Genes Fund – To Help Bring New Money, Interest and Champions to the Rare Disease Community

Their exists an inherent need to bring more money into the rare disease community to fund research.  Individual rare disease specific organizations work tirelessly to try and raise capital to fund various research projects, with the hope of bringing new therapies, treatments and cures to their communities children.

Recently Jonathan Jacoby, CEO RARE Project and Nicole Boice, Founder/President, RARE Project announced the development of the Global Genes Fund at the Genetic Alliance Meeting held in Washington DC.   Jacoby and Boice, presented the project to rare disease foundations, scientists, parent advocates and some news media during the research symposium breakout sessions.  It was the perfect setting to announce the development of Global Genes Fund because this meeting was focused on developing collaborative initiatives to help facilitate more successful partnerships and help move the needle for the rare disease community as a whole.

The announcement and discussion was being made on behalf of the team behind the Global Genes Project, and those parent advocates and organizations that have worked so hard to help build the cause related campaign for the rare disease community!  Boice stated, ‘The Global Genes Project represents a true collaboration, a program developed ‘by the rare disease community, for the rare disease community’.”

With the addition of the Global Genes Fund, this initiative will be able to provide a capture mechanism for rare disease research funding in two ways; Individuals within the general populace will be able to fund rare disease research and will be able to choose what ‘in their lifetime’ research they are interested in supporting.   This will be accomplished by building a consumer facing platform (modeled after the successful micro-lending portal, kiva.org), and by the marketing and press efforts to engage the broader public that is being developed by Global Genes Project.

Second, with increasing interest by corporations to support this community of millions, the Global Genes Fund will exist to accept donations from corporate supporters that see value in supporting a large group of consumers/constituents,  and then will distribute the contributions to those research initiatives being promoted within the Fund.

“The goal is to bring new eyeballs, new money and new champions to support this community in need,” Boice stated.  “This will not take the place of disease organizations efforts but will provide an additional way to capture and build new donors who may not have known about rare disease or ever had an opportunity to give to one previously.”  The Global Genes Fund will offer this platform and campaign at no charge to the participants, however there will be a small commitment by the disease groups to help ensure the success.

So far, there has been tremendous support and excitement from rare disease advocates who see the value in a mechanism to bring new capital to this very underfunded community!  Expectations for launch will be  World Rare Disease Day 2011.

Until then, RARE Project will be working with advisers and the Global Genes team to develop the criteria for inclusion in this fund.  Organizations interested in learning more should contact Nicole Boice at nicoleb@rareproject.org.

Official Shoe for Rare Disease Day 2011, Denim Keds?

Just sayin’, if we had an official shoe. This would be a good one. Hello Keds? Hello Gap?

// Catherine Calhoun

Blue Jean/Denim Inspired Bracelet, lovely way to raise rare awareness.

I love this blue jean/denim inspired bracelet, it reminds me of the ones from when I was a kid that we’d get at the beach in a natural color, tie on, keep on until it fell off from wear. I’ll be wearing this for rare disease awareness just as soon as it arrives! And then hopefully Ella and I can figure how to make some more like it.

//Catherine Calhoun

Global Genes Project, denim/blue jean bead bracelet.

Prescription Drug User Fee Act (PDUFA)

The Prescription Drug User Fee Act (PDUFA) allows the FDA to collect fees from drug makers that produce specific human drug and biological products. These fees help to support the new drug approval process. In general, applications for products that have been designated as a drug for a rare disease are exempt from this fee. The statutory authority for PDUFA expires in September 2012. At that time, new legislation will be required for FDA to continue collecting user fees for the prescription drug program (the renewal of this Act is called PDUFA V). The FDA will hold the first of many meetings to gather input in advance of the next PDUFA, giving stakeholders an opportunity to weigh in on user fees the agency charges for drug review and how those fees are used by the agency. The July 1 meeting is open to consumer advocacy groups, health care professionals and scientific and academic experts, the FDA says in a notice in the Federal Register.

An example of PDUFA’s relevance for families impacted by a rare disease diagnosis is the drug Acthar, marketed by Questcor, which is used in treating patients with infantile spasms (“IS”), a rare form of refractory childhood epilepsy, and opsoclonus myoclonus syndrome, a rare autoimmune-related childhood neurological disorder. http://www.acthar.com/

OPSOCLONUS MYOCLONUS SYNDROME
Opsoclonus Myoclonus Syndrome is an extremely rare auto-immune disorder that affects as few as 80 children a year in the United States. It is caused by a neuroblastoma, a cancerous tumor, or a virus. It is thought that antibodies in the immune system designed to fight off the tumor (or virus) end up attacking the brain as well. Opsoclonus” is an unusual disorder of eye movement in which both eyes dart involuntarily (dancing eyes). “Myoclonus” simply means brief muscle jerks and “ataxia” indicates incoordination. The syndrome has been called by many other names, such as “Kinsbourne syndrome,” “dancing-eyes-dancing-feet” or opsoclonus-myoclonus syndrome (OMS). Most children are less than two years old when diagnosed. There is no cure for OMS. http://www.omsresearch.org/ (A Global Genes Project Organization)

Facebook Groups—
Opsoclonus Myoclonus Syndrome Awareness
OMS Families – Opsoclonus Myoclonus

INFANTILE SPASMS – REFRACTORY CHILDHOOD EPILEPSY
The onset of infantile spasms (sometimes characterized as West Syndrome) is usually in the first year of life, typically between 4-8 months. The seizures primarily consist of a sudden bending forward of the body with stiffening of the arms and legs; some children arch their backs as they extend their arms and legs. Spasms tend to occur upon awakening or after feeding, and often occur in clusters of up to 100 spasms at a time. Prognosis is dependent upon the underlying disorder. Many underlying disorders, such as birth injury, metabolic disorders, and genetic disorders can give rise to spasms, making it important to identify the underlying cause. In some children, no cause can be found. http://www.tsalliance.org/

Infantile Spasms and TSC: A Devastating Diagnosis

From Twitter—
@bsangel: it’s not for a prize or anything, it’s just about spreading infantile spasms awareness. (http://bit.ly/bq46Zz)
@ PLM_Epilepsy: our journey with infantile spasms: epilepsy walk http://bit.ly/9kSIja

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REMINDERS:

• June 23, 2010: Congressional Hearing on the Food and Drug Administration’s (FDA) Review Process for Rare & Neglected Diseases
• June 29-30, 2010: Public Hearing Regarding Food and Drug Administration’s Review and Regulation of Articles for the Treatment of Rare Diseases
• July 1, 2010: Prescription Drug User Fee Act (PDUFA) Reauthorization Stakeholder Meeting

Facebook
Twitter

Meet Denim W. (and on the birthday of blue jeans).

From Beth W.

Just got your message about it being the birthday of jeans. My son’s name is Denim. Since the day he was diagnosed with Biliary Atresia our whole family has worn a patch of denim held on by a donor awareness ribbon. It’s a good conversation starter have got to tell a lot of people about BA and the importance of being a donor just cause they’ve asked why I wear a patch of denim.

Biliary Atresia is a liver disease. You can contact Beth W. if you would like to learn more (here).

// CC

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