On the Verge of a Cure: You Can Help

Imagine you are the parent of a child with a disease so rare, that it affects only 500 people in the United States.

You'd quickly find that "big name" diseases garner almost all the available research funding. And when every moment and every dollar counts, you might even start to give up hope.

They never give up hope at Scripps Research, thanks to generous friends like you. At Scripps, they are able to support brilliant scientists like Dr. Stephanie Cherqui – whose research is giving cystinosis patients and their families a reason to believe in a cure for this deadly disease.

Cystinosis is a rare genetic disorder that gradually destroys each and every organ in the body. Despite advances in treatment, children with cystinosis are plagued with diabetes, blindness, and kidney disease. Few patients survive beyond the age of 40.

Dr. Cherqui's research on cystinosis has been called the greatest prospect for a cure by many in her field, including the Cystinosis Research Foundation. Working in the Department of Molecular and Experimental Medicine at Scripps Research, her team has been making steady progress toward that goal.

In 2009, they used bone marrow stem cell transplantation to address symptoms of cystinosis in mice. The procedure virtually halted the process responsible for the disease. Now, just three years later, Dr. Cherqui's research has already moved into human clinical trials – an extraordinary jump that could provide insights on treating a number of other diseases affecting multiple body organs.

They simply would not be able to support breakthrough research like Dr. Cherqui's without the generosity of others. Please make a gift today and help them fund the basic biomedical research that leads to better treatment and cures.

If you or someone you love suffers from a disease – whether it's cancer, diabetes, or one that affects only hundreds of people – you know that scientific breakthroughs are critical.

Please join them in supporting scientists like Dr. Cherqui, who are invested in saving lives through their work.

Source: The Scripps Research Institute

Image Credit: http://www.stockfreeimages.com