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…
The National Organization for Rare Disorders (NORD) is the largest nonprofit independent organization for rare disease information, research, and advocacy. The organization is devoted to the treatment of all rare…
1. Rare Neuromuscular Disease Primary periodic paralysis (PPP) is group of rare neuromuscular diseases characterized by disrupted communication between nerves cells and skeletal muscles. PPP is a result of failures…
After over a decade, Debbie Zelman, founder of Debbie's Dream Foundation, succumbed to her stomach cancer at age fifty. From the beginning, Debbie's prognosis was not good. When the cancer…
According to The Pharma Letter, over the last 20 years progress with the development of orphan drugs for rare diseases has been made, but many question, is it enough? There…
On Christmas Day, Chris Bombardier, a man suffering from hemophilia B, traveled to Antarctica. While most of the people that go there scientists, Chris has a completely different goal in…
Daniel Downing received a personal visit from certain alien robots from outer space. The Transformers Autobots came to town to hang out with the 5-year-old terminally ill boy who has…
Originally reported by The Straits Times, the Chew family from Singapore does everything they can to keep their nine-year-old daughter alive, even if it means spending their life savings and…
According to a story on PRNewswire, the biopharmaceutical company, Enzyvant, kicked off their Farber Disease Natural History Study in October. Acid ceramidase deficiency, also known as Farber disease, is an obscure…
An international study conducted by researchers at the University of Copenhagen has discovered an essential mechanism that controls wound healing in the intestine. The new discovery shows that the cells…
Doctors from the Colorado University School of Medicine recently conducted research indicating that an exercise protein could help prevent the onset of Parkinson’s disease symptoms. Parkinson’s disease is a rare…
An engineering science professor from British Columbia designed an all new high resolution scanner that will enable eye problems to be diagnosed earlier. This will also allow earlier treatment and…
Orla Tinsley, an Irish-born journalist living in New York, has had to put her work in hold and is now on life support at New York-Presbyterian Hospital. As a sufferer…
A couple of days before Christmas, the FDA granted a regular approval to the drug, hydroxyurea (Siklos) for treatment of pediatric sickle cell anemia. Sickle cell anemia is a chronic…
At six years old, Benny was unable to sit up, roll over and scoot like other babies his age. Missing all the major milestones, his mother Jennie knew that something…
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