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…
Music is sometimes more powerful than we even realize on the daily. Music keeps little 6-year-old Ollie Carroll going, even though he understands his life will be short. Ollie and…
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…
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…
It's hard to find funding for rare diseases research. Patients and the people who love them don't have the same established networks of support that help people struggling with more…
When someone is diagnosed with a rare disease and it looks like their days could be numbered, it can often be difficult to cope. Many people simply don't want to…
According to an article at publicnow.com, the FDA has granted Breakthrough Therapy Designation to avelumab combined with INLYTA (axitinib) for people with advanced renal cell carcinoma. This designation should accelerate…
Good news for Prometic Life Science new drug application! The pharmaceutical company has been searching for a cure for idiopathic pulmonary fibrosis (IPF), a rare lung disease which you can…
According to a story from wect.com, Dr. Jorge Figueroa, who has been a teaching for nine years at the University of North Carolina at Wilmington, has developed a reputation as…
Denise West and Donna Crouch have much in common. It’s no surprise that they’ve become friends and grown to support one another. Perhaps what they have most common in common…
For Johanna Watkins, a woman from Minnesota, life can be like walking through a minefield. Ms. Watkins had mast cell activation syndrome. As a result, exposure to everyday experiences such…
Kendall and Ava are not related by blood. They do, however, have one amazing thing in common- something that saved their lives and defeated autoimmune hepatitis. Keep reading, or follow…
Ellie the stuffed Emesis Elephant recently did what most human beings could only dream of. She flew. To be clear she went skydiving to raise awareness for cyclic vomiting syndrome…
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