Gene therapy has successfully made its presence known in the medical field this last year, reported ABC News. Dr. Francis Collins of the National Institutes of Health isn't afraid to…
WASHINGTON, D.C. & SAN DIEGO — Children’s National Health System and Retrophin, Inc. (NASDAQ: RTRX) are pleased to announce the creation of the Retrophin Rare Disease Network at Children’s National.…
When you are going through the daily struggles of living with a rare disease, you feel alone. It is hard to accept the support from family and friends because they…
The Cystinosis Research Network (CRN) is offering scholarships to prospective college students who have been impacted by the condition. The organization offers two different scholarships, one for a student that…
I am always incredibly inspired by the people that don’t allow their rare diseases to prevent them from doing what they love. Whether it’s been Charity Tillemann-Dick with opera or Olivia…
Pondering “If only” and “What if “ adds anxiety for rare disease families. It seems to be a part of human nature that we do a lot of looking back,…
Have you been told that you had to stop doing your passion in life because of your rare disease? This is the story of opera lover, Charity Tillemann-Dick. Her life…
Welcome back, Patient Worthians! We're starting out the new year with stories of strength and awareness. This week, we have an article about a man with CSF leak, whose life…
Debora Chambless was on a steady decline. She suffers from nonalcoholic steatohepatitis, the most extreme form of non-alcoholic fatty liver disease. Debora and her husband Bill are active churchgoers, and…
No family faces life's unexpected challenges better than the family of Bella and Liam Brya. Allison and her husband, Bill, deal with the struggles of having two children, Bella and…
In 2009 Emily Rochac Argueta realized her vision was rapidly deteriorating headed towards blindness, reported Times Union. The aspiring physician assistant was diagnosed with keratoconus, a rare eye disease that…
High school football player McCaulley McGuire doesn't let his isovaleric acidemia keep him from playing the game, reported Times Free Press. Diagnosed with the rare disorder when he was only…
Nanette Whitfield, stood beside her best friend and husband as amyotrophic lateral sclerosis (ALS) painfully took his life, reported The Record Courier. Watching someone you love slowly lose everything has…
Despite the obvious cliché nature of the old adage, knowledge really is power. Technology and medical advancements give doctors and researchers more information than anyone could have fathomed even a…
Olivia Ports recently performed for over a thousand people, including the Mayor and Governor of Chicago, helping to raise $3.75 million for research by Lurie Children’s Hospital in only one…
Ryan Cole is a beautiful baby. You would never know from looking at him that he was struggling for life fighting a rare disease called biliary atresia, reported Courier Mail.…
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.…
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…
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…
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…
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…
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…
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…
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