Rare Disease Touches Family of Blogger Friend: GM-1 Gangliosidosis

NiceDeb’s post today is telling us about a very rare disease, GM-1 Gangliosidosis. Types I, II and III. Her young son has been diagnosed with Type II. Republican Congressman Cliff Stearnes (FL) has introduced legislation offering hope to the families dealing with a variety of nearly unknown diseases. In the case of GM-1 Gangliosidosis (all three Types) there is no treatment. She is asking us to contact Congress and encourage them to push these measures through. Find your Senator and Congressman/woman here.

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Nice Deb:

There is currently no cure or treatment for these diseases. In the United States,  research in the areas of enzyme replacement and gene therapy for GM1 gangliosidosis is ongoing but has not advanced to human trials. What’s the hold up? Why isn’t the research advancing? There’s an urgent need for a cure. The children who live with this disease are losing physical and cognitive abilities by the day. I know.

GM1 trials on humans are still years away.

There are currently some bills working their way through Congress that to help unlock lifesaving treatments:

HR 3737 the Unlocking Lifesaving Treatments for Rare Diseases Act
http://www.curetheprocess.org/ultra_act

ULTRA S 606/HR 3059 the Creating Hope Act [passed the House on May 31st, 2012]
http://www.congressweb.com/cweb2/index.cfm/siteid/KAKI/action/TakeAction.Cont…
(In “Affiliation” box write: National Tac-Sach and Allied Disease)

H.R. 4132: FAST Act

http://www.govtrack.us/congress/bills/112/hr4132

Congressman Cliff Stearns (R-FL), a senior member of the House Energy and Commerce Committee, says that “in 1992, the FDA created an Accelerated Approval process to make new drugs available earlier to treat serious diseases and fill an unmet medical need based on a surrogate endpoint. However, the modern FDA’s approval rate for drugs and medical devices has slowed immensely.  Last March, Rep Stearns along with Rep. Ed Towns (R-NY) offered H.R. 4132, the Faster Access to Specialized Treatments (FAST) Act. 
The following information is fromWikipedia:
Prior to passage of the Kefauver Harris Amendment in 1962, the average time from the filing of an investigational new drug application (IND) to approval was 7 months. By 1998, it took an average of 7.3 years from the date of filing to approval.[6]
“There aren’t many bills that pass the House that give instant hope to so many people in need.  This one does,” said Congressman McCaul, founder and chairman of the bipartisan Congressional Childhood Cancer Caucus.  Countless numbers of children and their families who are unable to treat their disease because of a lack of adequate treatments are counting on this legislation, as will many more children who may one day learn that they have a life-threatening illness.  The Creating Hope Act offers the best chance of encouraging pharmaceutical companies to develop treatments for children at no cost to taxpayers.”
Many of those diagnosed with GM-1 Gangliosidosis do not have 7 years to wait for an effective drug approval. Please put NiceDeb, her son and family on your prayer list, as well as those medical researchers capable of creating miraculous medicines, if our government simply gives them the opportunity. One day, it might be your child, grandchild, niece or nephew.
Linked at BadBluethe baddest news on the planet – thank you!

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  • Maggie you really are one of the nicest bloggers in the right-wing blogosphere. I can’t tell you how much I appreciate your help with this. Our son was diagnosed with educational autism at age 5, but it was always a questionable diagnosis. Last year I started suspecting that he was suffering from some sort of degenerative illness because cognitively and physically he seemed to be going backwards. It wasn’t until I asked his pediatrician to have his hips x-rayed, that we started down the path that led us to this diagnosis.

    At first I was relieved to finally have an answer. But then I was crushed, when I realized what his not so distant future held for him.

    Thanks again for helping us push for a cure. And thank God for those members of Congress who are working on the lifesaving legislation.

    • Deb, I have other legislators around the state I plan to contact and will try to keep up with whatever happens in the senate. I’m sure both Inhofe and Coburn would be a co-sponsor. Maybe Coburn, as a doctor, would sponsor. I noticed that Langford, along with Sullivan is sponsoring 3039. Maybe they’ve missed that there is companion legislation that needs their names on it.

      I can’t imagine how you are getting through this, but I know that we are given the strength to do what we must for our children, but nothing heals the heart, not even our deepest faith. You will stay in my prayers and I will do my best to keep up with legislative progress.

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  • NiceDeb and her son are in my thoughts and prayers.

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  • Grumpy, thanks for reblogging. I really appreciate it.