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

DANA POINT, Calif., Sept 01, 2010 /PRNewswire via COMTEX/ — 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

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/fund-hope-for-sick-kids 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 for which there are limited drug treatments. In the United States, 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.

SOURCE Children’s Rare Disease Network; The Global Genes Project

Interview: Amy & Chris Martin & Their Involvement with Duchenne Muscular Dystrophy

The following is an interview with Amy & Chris Martin who have a son with Duchenne muscular dystrophy. Amy and Chris have been instrumental in helping bring about the UCLA DMD Research and Clinic. Take a moment to learn more about how and why they are committed to bringing a cure to DMD.

How long ago was your son diagnosed?
3 years ago 

What was your reaction?
Shock, confusion and terror….like a bomb exploding on our house. 

What led you to Cure Duchenne?
Friends introduced me to Stan (Nelson) and Carrie (Miceli). Then I started doing research on the web and came across CureDuchenne. I liked the name.

We met Paul and Debra (Miller) and really liked their approach to finding and funding viable treatments that may help this generation of boys. We also liked the fact that they were in Southern California. There was no need to reinvent the wheel and start our own foundation. We wanted to work together.

What made you decide to get involved in fundraising?
Fundraising is the only thing we can do! We are not scientists. We are not doctors.  We are parents who are very involved in our community and like to give back. When we needed help we got it because of that community spirit. Scientists need money and we can help. Why wouldn’t we?

You’ve done very well with Dealing for Duchenne Celebrity Poker Tournament. How did you get started with such a successful event? (What’s your secret?)
Both Chris and I are very active within our community and at our kid’s schools. We volunteer and coach teams and always help friends in need. When we asked people for help in putting together an event, we got it!  That’s what friends and people in your community are supposed to do for one another.

We feel very blessed to have such wonderful friends. I guess the secret is being a good friend, and also being able to ask for help. People want to help but you have to ask for specific things. No one knows what they can do, you need to tell them.

What would you say to parents who’ve just received the diagnosis?
First I would say, in the beginning it is okay to do nothing. To feel sad, angry, and depressed. It helped me to learn as much as I could about the disease and to also talk to my friends. I didn’t want to get involved in parent support groups. I wanted to live life in the present and do whatever I could to make a difference in my son’s future.  

Everyone handles it differently. There is no right way to grieve. It is a process and ours is especially difficult. It is a burden to live with knowing that your child is dying inside and that there is nothing doctors can do to prevent it. We are not equipped to deal with that.

I cannot explain how hard it is to live with this burden of knowing. It doesn’t get easier, but you do get better at it. Denial is okay sometimes. I do not blame or judge anyone who wants to go away and live life with their child and wait for scientists to find something that will help. Sometimes I want to do that and sometimes I take time to do just that! But I know that the science needs funding, and I have to help – it is the only proactive thing I can do to give my son a fighting chance.

What would you say to people looking to get started with fundraising?
Ask your friends for help! Think about who you know, what they may be able to do to help you, and then ask them. Learn about the disease process, the research being done, and think about why no one knows about Duchenne. Talk about it. Be honest with your friends.

I know that every parent in my situation would do the same thing because we all fight for our kid’s futures every day. We all want to give our kids the best chance at living a full life. I never knew how to throw a gala or charity event. I asked for help. I was honest in saying “I don’t know how to do this, can you help me?”

If you want to do something talk to your friends about ideas, ask them for help, and you will be surprised. CureDuchenne is a great resource and ready to help any parent that wants to get involved.

Any other thoughts you’d like to share with the DMD community?
People need to know what Duchenne is. I have been in the awkward situation of hearing about children in my community who are suffering from Leukemia, cancers, diabetes, and other childhood diseases.

Everyone feels so sorry and sad for them, and yet I want to scream, “I would give anything for a doctor to tell me that my son has an 80% percent chance of survival if we do a horrible (but life saving) treatment for 2 years!” I’d take even a 70% or 50/50 or anything!

Duchenne is a death sentence. So were AIDS, Cancer, Diabetes, and Cystic Fibrosis. If we can’t talk about how bad it is, we will never get enough attention or funding.

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Acceleron Pharma Receives FDA Fast Track Designation for ACE-031 for the Treatment of Duchenne Muscular Dystrophy

CAMBRIDGE, Mass.– August 19, 2010 – Acceleron Pharma, Inc., a biopharmaceutical company developing novel therapeutics that modulate the growth of cells and tissues including muscle, bone, fat, red blood cells and the vasculature, today announced the United States Food and Drug Administration (FDA) granted orphan designation for ACE-031 for the treatment of Duchenne Muscular Dystrophy (DMD), a fatal neuromuscular disease in which patients experience a progressive loss of muscle mass and strength. ACE-031 is an investigational protein therapeutic being developed to increase muscle mass and strength.

“In the past two weeks, the FDA has granted both orphan status and Fast Track designation to ACE-031 in recognition of the drug’s potential to address the enormous unmet medical need for therapeutics to treat DMD patients”, said Matthew Sherman, M.D., Chief Medical Officer at Acceleron. “We will continue to work collaboratively with clinical investigators, health authorities and patient advocacy groups around the world to develop ACE-031.”

Orphan drug designation is granted by the FDA Office of Orphan Products Development to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases and disorders. Under the Orphan Drug Act, the FDA may provide grant funding towards clinical trial costs, tax advantages, FDA user-fee benefits, and seven years of market exclusivity in the United States following drug approval by the FDA. The approval of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval. For more information about orphan designation, please visit the FDA website at www.fda.gov

About ACE-031

ACE-031 is an investigational protein therapeutic that builds muscle and increases strength by inhibiting signaling through a cell surface receptor called activin receptor type IIB (ActRIIB). ACE-031 is a recombinant fusion protein that is produced by joining a portion of the human ActRIIB receptor to a portion of a human antibody. This creates a freely circulating, decoy version of ActRIIB which removes proteins, such as GDF-8 (myostatin), that limit the growth and regeneration of muscle. Animal studies with ACE-031 suggest that blocking signaling through ActRIIB leads to increased muscle mass and improved physical function in a range of animal models of muscle disease, including models of muscular dystrophy. ACE-031 is currently being studied in a phase 2 clinical trial of patients with Duchenne Muscular Dystrophy (DMD) who are also receiving corticosteroid therapy.

About Duchenne Muscular Dystrophy (DMD)

Duchenne Muscular Dystrophy (DMD) is a debilitating and fatal genetic disorder characterized by the progressive loss of muscle strength and function. It primarily affects boys and occurs in approximately 1 in every 3,500 live male births. DMD is caused by genetic mutations that result in the absence or a defect in dystrophin, a protein necessary to maintain the structural integrity of muscle fibers. This condition leads to deterioration of and damage to skeletal muscles, which eventually become infiltrated by non-functional scar and fatty tissue. As a result, patients experience a relentless decline in muscle strength that impairs their ability to walk, breathe and live independently. Many patients spend the majority of their lives confined to wheelchairs and eventually lose all upper body function. Few patients survive beyond their late-20s when their heart and respiratory muscles weaken and eventually fail.

About Acceleron

Acceleron is a privately held biopharmaceutical company committed to discover, develop, manufacture and commercialize novel biotherapeutics that modulate the growth of red blood cells, bone, muscle, fat and the vasculature to treat musculoskeletal, metabolic and cancer-related diseases. Acceleron’s scientific approach takes advantage of its unique insight into the regenerative powers of the TGF-β superfamily of proteins. ACE-011 is in Phase 2 clinical trials in patients with anemia. ACE-031 is being studied in a Phase 2 clinical trial in patients with Duchenne Muscular Dystrophy and ACE-041 is being studied in a Phase 1 clinical trial in patients with advanced cancer. In addition, the company is developing several other new product candidates that increase muscle mass, control angiogenesis, inhibit fat accumulation and increase bone mass and strength. Acceleron utilizes proven biotherapeutic technologies and capitalizes on the company’s internal GMP manufacturing capability to rapidly and efficiently advance its therapeutic programs. The investors in Acceleron include Advanced Technology Ventures, Alkermes, Bessemer Ventures, Celgene, Flagship Ventures, MPM BioEquities, OrbiMed Advisors, Polaris Ventures, QVT Financial, Sutter Hill Ventures and Venrock. For more information, visit www.acceleronpharma.com

CONTACT:

Acceleron Pharma:
Steven Ertel, 617-649-9234 
Vice President, Corporate Development

Paul Kidwell (Media)
Suda Communications LLC
617-296-3854

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