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.
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
ULTRA S 606/HR 3059 the Creating Hope Act [passed the House on May 31st, 2012]
(In “Affiliation” box write: National Tac-Sach and Allied Disease)
H.R. 4132: FAST Act
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.
“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.”
- 23 June 2012 at 1:06pm
- Before It's News 23 June 2012 at 2:06pm
- Help Needed in Unlocking Lifesaving Treatments for Rare Diseases (Video) « Nice Deb 23 June 2012 at 4:06pm
- Maggie’s Notebook | Grumpy Opinions