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.…
Baseball pitcher Tyson Ross recently signed to a Minor League contract with the San Diego Padres. In the past, Ross pitched for the Padres from 2013-2016. Now thirty years old,…
Batu Biologics just raised $1.3 million in order to continue funding the development of ValloVax, an immunotherapy treatment for cancer tumors. The crowdfunding project was started in May 2017. Initially,…
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…
Anna Gordon from West Virginia doesn't let her Friedreich ataxia define her, and she encourages anyone with a rare disease to not let it define them either, reported Public Broadcasting.…
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,…
The pharmaceutical company Protalix Biotherapeutics, Inc. announced that a drug currently in development called PRX-102 has been granted orphan drug status by the European Commission. The treatment is being developed…
The FDA passed along their nod of approval for the sought after gene therapy treatment, Luxturna, reported Engadget. This will be used to help aid those made blind by a…
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…
DelMar Pharmaceuticals announced that its flagship cancer treatment drug, called VAL-083, was given "Fast Track" status by the Food and Drug Administration. VAL-083 is a possible treatment for a type…
Cori Salchert is a hero. Recently featured on the Today Show, Cori was honored for being the ultimate supermom, taking in foster children who are sick with terminal diseases, report…
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…
Human beings have always speculated about the future. Science fiction is probably one of the most prominent examples, and stories are replete with robots, lasers and flying cars. But no…
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…
It's been 35-years since the passing of the Orphan Drug Act, and creation of the National Organization for Rare Disorders (NORD), reports PR Newswire. Even though these organizations recognize there…
According to a story from tennessean.com, Trishia Warrick, a Michigan native, moved down to Tennessee when she was in eighth grade. Sadly, she never felt very welcome growing up in…
Jacob Engram, a twenty-one year old high school student with Apert syndrome, became the first junior officer for the city of Vicksburg. Read more about this story in The Clarion. Apert…
According to a story from Pet Age, a combination of business leaders, pet owners, and reptile hobbyists came together and raised $45,000 for the grieving family of Anna Hupp, who…
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…
Classifying Brown-Vialetto-Van Laere syndrome (BVVL), also known as Riboflavin Transporter Deficiency (RTD), as a rare disease in an understatement. There have only been about 100 cases ever diagnosed. That means…
Rare diseases sufferers are the unsung heroes who often fall into the shadows behind more common cancers, but some scripted and reality shows are casting a spotlight on them. In…
© Copyright 2024 Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
