Neuroendocrine tumors are growths which form from neuroendocrine cells in various places throughout the body. They can occur in the gastrointestinal track, the pancreas, the lungs, the thymus, and other…
Biobanks store biological samples to help scientists research certain diseases. Registries collect information from patients and standardize it, but no samples are stored. Which of these is best for rare…
Batten disease is a lysosomal storage disorder. It's neurodegenerative, meaning it results in the deterioration of neurons. One of the most common forms of this disease is caused by mutations in the…
According to a story from University at Buffalo, a recent discovery of a new genetic mutation continues to build on decades of research from Dr. Richard Gronostajski, a professor and biochemist…
According to a story from the Australian Jewish News, a Jewish teen named Gidon Goodman was born with Gaucher disease. Recently, he started the first Gaucher disease patient support group…
According to a story from Raconteur, finding sufficient funding for the development of new therapies for rare diseases has always been a major challenge. Rare diseases are defined in the…
According to a story from tradeshownews.vporoom.com, the biopharmaceutical company Viking Therapeutics, Inc., has recently released the results from a Phase 2 clinical trial. This trial is testing the company's investigational therapeutic…
According to a story from Benzinga, the drug developers AstraZeneca and Merck have recently announced that the US Food and Drug Administration (FDA) has decided to accept a supplemental New…
According to a story from nature.com, Amber Sapp's twelve year old son Garrett had been participating in a clinical trial that was testing a new potential treatment for his Duchenne…
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