When a patient is told that they have a life-threatening rare disease and then told that there is no cure, every effort is made to attend to their physical needs.…
The Orange County Guide features doctors’ articles in each issue. Dr. Justin West’s search to cure his son’s rare disease will be familiar to families also in search of…
Vanderbilt University has just announced a new collaborative agreement with Soleno Therapeutics in aim to develop KATP channel activators as a therapeutic for rare diseases. This therapy has already been…
As reported in News Wise, these oncologists don’t like their options. They want to provide patients with the best possible options to tackle the lethal cancer mesothelioma, but their hands…
Together, Takeda Pharmaceuticals and the Children's National Hospital have created a program that aims to create and improve a standard of care for rare disease patients. As these patients often…
According to a story from Market Watch, the gene therapy company AVROBIO, Inc. has recently announced that its investigational gene therapy candidate AVR-RD-04 has earned Orphan Drug designation from the…
Each year, over 200 million people worldwide, many in Africa and South Asia, are diagnosed with malaria. Unfortunately, this mosquito-borne illness is also fatal for thousands. While some countries are…
A recent study has documented the differences between two rare forms of childhood brain inflammation. The two similarly presenting conditions are often confused for one another. A better understanding of…
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