My name is Melissa Klein. I am 21 years old and I was diagnosed with Mitochondrial Neurogastrointestinal Encephalopathy (MNGIE) in December 2012. MNGIE is an autosomal recessive disorder that has no known cure. It affects the digestive and nervous system, including symptoms such as:
- hearing loss
- peripheral neuropathy, leukoencephalopathy (white matter)
- dysphagia (trouble swallowing)
- nausea, vomiting
- diarrhea, bloating, abdominal pain
- intestinal blockage
- extreme weight loss
- muscle weakness, reduced muscle mass
I have developed all of these symptoms, and my quality of life is slowly getting worse. They say MNGIE patients usually pass on in their mid-30's, but I have been told that I won't make it that far.
I have been to NYC to see Dr. Michio Hirano, back in January, to see if he could help me with his clinical trial. However, because I also suffer from diverticulitis, I was considered high risk and ineligible and could not participate. Hirano informed me that there was being more research done in London, and it would more than likely reach the states in a few years, but after contacting Dr. Bax, I feel like this is my last chance, and I need to have this treatment done as soon as I can!
What We Need & What You Get
Research is being done and clinical trials are being held, however funding is scarce. The treatment I am working towards is called erythrocyte encapsulated thymidine phosphorylase (EE-TP) and is designed to "fix the molecular defect behind MNGIE by replacing thymidine phosphorylase, the deficient enzyme, by encapsulating it within the patient's own erythocytes in vitro, and then returning these to the patient."
This treatment is being given compassionately, meaning the patients must provide funding themselves in order to pay for it. Two patients (that I am aware of) have received this treatment, and have both experienced amazing results. The cost of EE-TP would be about $6,100 a month, and this campaign is meant for one month and the travel costs for my husband and I.
When you donate, not only are you doing me a huge favor, but you are saving my life. I don't have much, but I would love to say thanks by providing a few perks that are all unique and very personal. If I could pay each and every one of you back, I would do that and more.
All custom perks will be discussed via email, since that's the easiest way to contact me.
Please email me at email@example.com
If I were to reach my goal, I would be able to travel to London with my husband, meet Dr. Bridget Bax, and be able to undergo the EE-TP treatment that could slow down the progression of my disease and prolong my lifespan.
Other Ways You Can Help
I know my goal is high, and I know that with today's economy, not everyone can donate to a cause like mine. And even though every dollar helps, it would also help me immensely if you spread the word! Some ways you could help me by only spending a minute or two of your time are:
- Share Share Share! Share my cause with everyone you know! Use indiegogo's share buttons!
- Help me spread awareness!! MNGIE is a very rare mitochondrial disease. Educate yourself and your friends and family, and get as involved in as many events as you can.
- Visit my blog and Facebook page for more information. Share those pages, too!
Team on This Campaign: