Imagine having two daughters who both have a rare disease and watching them struggle — to be understood, to remember things, to learn, to be comfortable in social settings, to follow one-step directions, to be able to bond with friends and family. Now, imagine a clinical trial showed promise for your older daughter. You notice a remarkable difference in her daily living, and suddenly, your home is more peaceful, and she is starting to thrive. The treatment is potentially life-changing. You now have hope for your older daughter and her younger sister. But then, the medicine is potentially at risk of not even being reviewed by the U.S. Food and Drug Administration (FDA), let alone app
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