WASHINGTON (AP) — Good news for nearly 30 million Americans with a rare disease: Drugmakers are doing much more research on rare, often-deadly diseases with few or no treatment options.
They’re running patient tests of more than 5,400 potential new medicines, many for multiple conditions. Nearly 1,800 research projects are for rare diseases. Hundreds more are for disorders with no new treatments in many years.
That’s according to a study being released Thursday by the trade group Pharmaceutical Research and Manufacturers of America.
It found that thousands of those experimental drugs could be the first in a new medicine class with a unique target or effect. Nearly 600 use new technologies, from gene therapy to cloned antibodies, to precisely target the disease site. Others are for tiny subgroups of patients with genetic variations.
How about finding a cure for cancer??? To many people die every year because of cancer and millions of dollars are raised to find a cure but no cure yet!
I’m a potential beneficiary.. I have a genetic disorder that causes blindness and run the Choroideremia Research Foundation based here in Springfield. We’re funding Pre-clinical work at UPenn that will lead to human genetic therapy trials hopefully by this summer. The science is amazing and I’m praying it will be in time to save what little sight I have left!