RareArtist.org – Rare Art Created by Rare People to Support Rare Disease Awareness

What a great idea — a new website to showcase Rare Art created by Rare People to support Rare Disease awareness.  The Kakkis Everylife Foundation has launched RareArtist.org for artists of all ages affected by a rare disease.

There are almost 7,000 rare diseases that affect more than 25 million Americans — many of them are children like Addi and Cassi who suffer from Niemann Pick Type C disease which causes childhood dementia and is fatal.

The EveryLife Art Contest was established to empower artists affected by Rare Diseases to express their unique struggle with a rare disease.  An art competition is being held and is open to all artists affected by a Rare Disease ages 5 and older.

Check out RareArtist.org today and upload your art!

Social Security Disability ‘Compassionate Allowance’ – Only For Those Who are Impoverished?

Today I called the Social Security Office in Nevada to find out about the compassionate allowance program for our twins. Last month, the Social Security Administration added 38 more conditions to the already 50 rare diseases and cancers designated for compassionate allowance, which provides expedited review of disability applications from people with severely disabling conditions. Niemann Pick Type C disease was added to the list and after reading the press release I assumed our children would qualify.

After navigating  through the Social Security automated voice activated phone system for 30 minutes and losing the connection, I finally managed to get through to a live person. In the State of Nevada, you can select a Social Security appointment by telephone or in person. I chose the telephone appointments. Apparently, we can’t set up back to back appointments so I made them for March 18th and 19th at 10:32am.  All appointments in our State are done at 2 minutes after the hour. Only the government could come up with such a thing.

I was told that each appointment would be 1 ½ hours long to fill out the information. I tried to explain that our applications will be identical except for one minor change – one application will say Addison and the other Cassidy.  Still two appointments (unless I decide cancel — I’ll get into this below).

Social Security had some quick questions for me – how much is our annual income (I could not recall the number on the phone) and do Addi and Cassi have siblings (No).  They also wanted to know if I am “working.”  I am not officially working a job outside the home but I do spend all my time “working” to find treatments for our girls and we spend a significant portion of our money/income on funding research and experimental treatments.  The call was all very vague but I made the appointments to get real facts on the program in order to share with others in my situation.

A friend of mine who has a child with a different lethal rare disease told me this morning that with her husband’s unemployment and her part-time job, they make the ‘big bucks’ and don’t qualify for the program. I was shocked. I guess you have to be completely impoverished to get any assistance — little to no income from what I understand.  I now want to find out the truth as to who gets what so that people are not wasting their time pursuing something they obviously don’t qualify for. If a family with three kids (one who is dying) and who are on unemployment and working part time jobs don’t qualify, who does?

When a major announcement is made in the media touting how the government is helping kids who are dying, they might want to put in a caveat that says, “you can only qualify if your parents make X amount per year.”  I am looking at spending 3 hours of my valuable time to find out we make too much money.  I am certain we are making too much money but the way they promoted the program made it appear that all qualify.  How many people is this program truly helping?  And why would they want to accept and process two applications from me?  What a total waste of government time and resources!

For many people applying for benefits, the Social Security Disability process is a slow one. Being awarded benefits can take many months, often years. I was told our case would be reviewed within 20 days of filing the applications. A typical time frame for review is 120 days. I was told the “child rate” in the State of Nevada is $674 (this is the maximum amount per child).  Who could live off of $674 a month and why not 675?

The National Organization for Rare Disorders (NORD) helped get the list expanded. I am going to contact NORD to see if they know what the income limitations are for this program and why the income number was not put into the press release.

If you have a rare and terminal disease and want to find out about the compassionate allowance program, click here to go to the SSA website.  They won’t tell you if you qualify. I suspect most people don’t.  I found a helpful State-by-State list of Social Security Offices on a Huntington’s disease website if you wish to pursue it.

In addition to Niemann Pick Type C disease, some of the newly added conditions for compassionate allowance include Ataxia Telangiectasia, Hurler Syndrome Type IH, Idiopathic Pulmonary Fibrosis, Neonatal Adrenoleukodystrophy, Sanfilippo Syndrome and Wolman disease.

Here is a quick list of the new Compassionate Allowance conditions:

• Alstrom Syndrome
• Amegakaryocytic Thrombocytopenia
• Ataxia Spinocerebellar
• Ataxia Telangiectasia
• Batten Disease
• Bilateral Retinoblastoma
• Cri du Chat Syndrome
• Degos Disease
• Early-Onset Alzheimer’s Disease
• Edwards Syndrome
• Fibrodysplasia Ossificans Progressiva
• Fukuyama Congenital Muscular Dystrophy
• Glutaric Acidemia Type II
• Hemophagocytic Lymphohistiocytosis (HLH), Familial Type
• Hurler Syndrome, Type IH
• Hunter Syndrome, Type II
• Idiopathic Pulmonary Fibrosis
• Junctional Epidermolysis Bullosa, Lethal Type
• Late Infantile Neuronal Ceroid Lipofuscinoses
• Leigh’s Disease
• Maple Syrup Urine Disease
• Merosin Deficient Congenital Muscular Dystrophy
• Mixed Dementia
• Mucosal Malignant Melanoma
• Neonatal Adrenoleukodystrophy
• Neuronal Ceroid Lipofuscinoses, Infantile Type
• Niemann-Pick Type C
• Patau Syndrome
• Primary Progressive Aphasia
• Progressive Multifocal Leukoencephalopathy
• Sanfilippo Syndrome
• Subacute Sclerosis Panencephalitis
• Tay Sachs Disease
• Thanatophoric Dysplasia, Type 1
• Ullrich Congenital Muscular Dystrophy
• Walker Warburg Syndrome
• Wolman Disease
• Zellweger Syndrome

Rare Disease Idea Leads ‘Ideas For Change In America’ Competition by Change.org

Ideas for Change in America is a competition that empowers citizens to identify and build momentum around a diverse range of ideas for addressing the challenges our country faces.

From January – February 2010, more than 2,500 ideas were submitted and voted on by 100,000 people across the country. The top three rated ideas from each issue category qualified for the final round.

Right now, 25 Million+, It is time to care about rare disease is rated as the #1 overall topic! Let’s help it stay at the top.  Vote here!

The 10 most popular ideas will be presented to relevant members of the Obama Administration, and Change.org will subsequently mobilize its full community to support a series of grassroots campaigns to help turn each idea into reality.

Please take a few minutes and cast your vote on behalf of the 30 million Americans affected by rare disease — that’s 1 in 10 people in the US living with a chronic or life threatening rare disease!   It’s time to care about rare!

FDA and EMA Forge Rare Disease Collaboration; Announcement Coincides with World Rare Disease Day 2010

In recognition of World Rare Disease Day 2010, the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) announced that they will collaborate together and now accept a single annual report from sponsors with an orphan drug designation for rare medical conditions.

The joint announcement was made by Dr. Timothy Cote, director of FDA’s Office of Orphan Products Development and Dr. Jordi Llinares, head of Orphan Medicines at the EMA and marks an important step forward towards increasing data sharing between the two agencies.

An ‘annual report’ for an orphan drug is information that is typically provided about the development of orphan medical products, including a review and status of ongoing clinical studies, a description of the investigation plan and anticipated or current problems in the process that may impact an orphan product designation.

The submission is voluntary and applies only to sponsors who have obtained an orphan designation status for their product from both the FDA and EMA. Each regulatory body will conduct their own review and assessment of the annual report to assure the information meets all the legal and scientific requirements of each agency.

By allowing a single annual report submission to both regulatory agencies, the paperwork process is streamlined and precious time is saved as organizations can focus their energies on moving drug development forward instead of duplicating paperwork efforts. The FDA and EMA plan to exchange the annual reports electronically through a secure portal starting Feb. 28, 2010.

Hopefully, we will see more of this type of collaboration in the future by both agencies.  I am looking into filing my Orphan Drug Application for Cyclodextrin with the EMA and I am happy to see the two agencies working more closely together.

Denim Blue Jeans Ribbon To Symbolize Rare Disease Awareness; New Campaign of Jeans and Genes A Perfect Fit!

The Rare Disease community now has a powerful and universal new symbol to rally around — a blue jeans denim ribbon created by the Global Genes Project and the Children’s Rare Disease Network.  The worldwide initiative lauched today and is focused on raising awareness for millions of children living with rare and debilitating diseases like Addi and Cassi.  

Just like the pink ribbon symbolizes breast cancer awareness and the red dress created by the American Heart Association symbolizes heart health, the denim jeans ribbon is a perfect fit to symbolize the countless illnesses that involve gene defects like Niemann Pick Type C, Cystic Fibrosis, Prader-Willi, and Fragile X.

Numerous Corporations, Non-Profit Foundations, Research Institutions, Hospitals and Advocacy Groups are supporting this new demin campaign for rare disease.  Below is the press release announcing the initiative which is just in time for World Rare Disease Day 2010.

DANA POINT, CA – February 1, 2010 – Hope – It’s In Our Genes.™  The Children’s Rare Disease Network (http://www.crdnetwork.org) today announced the Global Genes Project (http://www.globalgenesproject.org), an initiative designed to raise awareness about the prevalence of rare diseases that afflict millions of children worldwide. Rare disease advocates organizing the campaign selected a blue jeans denim ribbon as the international symbol of hope for children suffering from life threatening and chronically debilitating rare diseases.

More than 90 corporations, non-profit foundations, research institutions and children’s hospitals have signed on to support the Global Genes Project including: 23andme, Amicus Therapeutics, Better Health, BioMarin, CheckOrphan, CollabRx, Counsyl, Coriell Institute, DonateGames.org, FasterCures, FreeForm, Genetic Alliance, Geni.com, Genzyme, Hide and Seek Foundation, Jackson Laboratory, Kakkis EveryLife Foundation, Knome, Lili Claire Foundation, Life Technologies, Mayo Clinic and Shire.

Numerous rare disease foundations from countries including Australia, Argentina, Belgium, Canada, China, New Zealand, Philippines, the United Kingdom and United States are helping support the global movement by organizing activities throughout February and leading up to World Rare Disease Day 2010.
“The Global Genes Project is providing a new platform to help unite people under a common goal and to make a difference for all children suffering from rare disease,” said Nicole Boice, Founder, Global Genes Project. “We want to create a world where children suffering from chronic disease have equal access to life saving drug treatments.”

Growing Public Health Concern; Inequity Exists

Rare disease is an important and growing global public health concern that can no longer be ignored. While each disease may be rare when considered by itself, rare disease collectively impacts the lives of millions. According to estimates from the National Institutes of Health (NIH), approximately 6,800 rare diseases exist, many of which are genetic in origin, translating to an affected population of 25-30 million Americans or approximately 1 in 10 people. In Europe, the number affected is almost identical. Approximately 75% of those affected by rare diseases are children, making the rare disease category one of the most deadly and debilitating for children worldwide.

Despite the success of the Orphan Drug Act of 1983, pharmacologic treatments exist for only a small fraction of rare diseases, or approximately 200 of the 6,800 rare diseases. After 25 years of orphan drug regulations and legislation, the development path to approve new drugs has not stimulated orphan drug development for small populations of patients facing rare disease.

Current studies also suggest that it takes more than a dozen years and hundreds of millions of dollars to take a potential drug from discovery to the marketplace. As a result, severe inequity exists in access to care and treatment for millions of rare disease patients.

Denim Blue Jeans Ribbon Campaign

Leading up to the annual World Rare Disease Day on February 28, 2010, the Global Genes Project will help facilitate a series of denim focused awareness events, charitable activities and engage corporate sponsors, government officials, celebrities and the media to help spread its lifesaving message of hope.

“Blue is the color of strength and our blue jeans denim ribbon is an ideal symbol that expresses solidarity,” added Boice. “Genes and jeans are a natural fit – both are universal, come in pairs and are unique to the individual. It’s a simple concept that anyone can embrace and a powerful way to raise awareness for rare disease.”

The Global Genes Project is asking rare disease advocates and supporters across the world to wear denim jeans and blue jeans denim ribbons the week of February 22 and leading up to World Rare Disease Day 2010. The organization is also asking supporters to display the blue jeans denim ribbon symbol on their websites and to actively invite co-workers, friends and family and their community to join in spreading this global message.

Blue jeans denim ribbons can be obtained by visiting www.globalgenesproject.org. Denim jeans and ribbon wearing supporters can submit digital photos on Facebook: http://www.facebook.com/group.php?gid=214263320931&ref=mf. To join the growing list of supporters, visit http://www.globalgenesproject.org/sponsors.php.  Global Genes Project Twibbons can be found here.

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