Happy New Years, Patient Worthians! New year, new you... new treatment news? This week, we have an article about a molecule that could change the way we treat cystic fibrosis.…
When 24-year-old Andy Pallone finally met Santa Claus, it heart-warmed his mother, and unexpectedly the rest of America, reported Belleville News-Democrat. His mother, Jennifer Pallone, wanted to capture the moment…
Pharmaceutical company, Santen, has been developing a drug, intravitreal sirolimus, to treat noninfectious posterior segment uveitis. Uveitis is a type of eye inflammation, in which the uvea, which is the middle part…
The more scientists learn about gut bacteria, the more fascinating it becomes. It can affect your mood, the foods you crave, and according two new studies, it can even change…
While April Archote faces the physical pain and financial hardships of living with a rare brain disease, she continues to be positive and count her blessings, CCHeadliner reported. In 2013, April was…
Sickle cell disease causes red blood cells to form abnormally, which disrupts oxygen function. Cancer Prevention Pharmaceuticals have been working with Vanderbilt Medical Center and NCI to start a new Phase…
A new study from researchers at UCSF found that a simple tool could optimize kidney treatment in children, by reducing dialysis and increasing pre-emptive kidney transplants. The tool is called…
There rare disease world has another rising hope in research and development. Her name is Christina Waters, and she is the founder and CEO of Rare Science, a California non-profit…
It's been two years since the Zika outbreak and now, two-year-olds born with microcephaly due to the infernal outbreak, are still suffering through long-term problems. Children who developed health problems…
Ionis and Roche have licensed together for the development of IONIS-HTTRx, a breakthrough drug in the treatment of Huntington’s disease. Huntington’s disease is a rare neurological condition that attacks the…
NORD, the National Organization for Rare Diseases, sent out their happiest wishes for the holidays, their utmost gratitude for 2017, as well as their plan of attack for the New…
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