Vertex Pharmaceuticals announced in late August that the U.S. FDA accepted applications of tezacaftor/ivacaftor for patients with cystic fibrosis (CF). Specifically, this combination treatment can be used in patients ages…
Spend a day (or two) in Boston indulging your inner film fanatic and watching a compilation of rare disease films from across the globe! Many films are short and sweet…
Natalie Stokes was always the curvy one in her group of friends. But when she started putting on more and more weight despite cutting down to only 1,000 calories a…
It's Fall "Y'all"! This week, we have PW Contribution from a man with CMT. We also have one on raising a child with rare disease. We also have good news…
Amber and Ryan Dollinger were preparing for the birth of their second son just as Hurricane Harvey was plowing toward them dead-on. They fled their Beaumont, Texas home and found…
This is the story of Eddie and Sarah Yang, their children, and challenges they face with a rare disease called congenital central hypoventilation syndrome (CCHS). Eddie and Sarah married in…
According to a lovely post on the International Waldenstrom's Macroglobulinemia Foundation website, Julianne married the love of her life at the age of 19; her husband John was 30. In…
Neuroblastoma has been one of those rare diseases that affects the youth and nobody messes with our future generation! This form of cancer weasels its way and burrows deep in…
Zika! It was the talk of the town during 2015-16 and has thus quieted down, but the virus still lingers and scientists are still working to combat it. A quick…
When raising children, parents spent a lot of time discussing the challenges of child-rearing with others, reading articles and books, and wondering if we were doing the right thing in…
Des Moines news anchor, Elizabeth Klinge, is receiving quite the social media buzz after posting heart-warming photos of her daughter Hanalay and her notable port-wine stain. The photos garnered a…
Welcome back Patient Worthians! We are back this week from the 2017 Global Genes Rare Advocacy Summit. Did you attend? If so, what did you learn? Let us know here. This…
Come to Canada to learn and talk more about acromegaly among specialists, professionals, advocates, patients and spouses! COST Acromegaly Patients & Spouses: FREE For all other attendees: $200.00 no refund…
The love of creating radio is something that can bring people together, according to the Riverine Herald. Whether it’s the perfect mix of melodies or the desire to connect listeners…
In Ancient Greece, you had to be part divine to be called a hero. Achilles' mother was Thetis, a rather minor goddess. Heracles’ father was a god you might have…
How does one raise awareness? Shouting on the street corner might work for a few hundred, or maybe even a few thousand, depending on how busy the corner is that…
When my son was eight years old he joined the swim team. For two hours a day, five days a week, he splashed around the pool mastering the Australian Crawl…
POW! BANG! KAPOW! On a typical day, Thor, Batman and Wonder Woman focus on purging the world of archenemies that threaten our very peace and prosperity. And the way things…
Scratchy jeans. No. 2 pencils. Trapper Binders. Squeaky shoes. Floor polish. Helpful bus drivers. Smiling teachers. Ah, the scents, sights and sounds of the first day of school. I loved…
What do you do when you are facing the unknown? We all have to at some point. And many of us will face the unknown while sitting in a doctor’s…
Causation vs. correlation. What does that even mean? If you ask your friend who majored in philosophy for a semester-and-a-half in college, you’ll probably get an explanation that sounds more…
Good news for patients suffering from neurological disorders that may have taken away their ability to walk. Multiple sclerosis, cerebral palsy, stroke and other diseases can often lead to paralysis.…
Everyone knows that businesses compete. If two businesses are in the same market, they have to compete, right? If you choose to buy a Ford, then that means you didn’t…
Amicus Therapeutics is an American therapeutics company based in Cranbury, New Jersey. They're new groundbreaking treatment announcement, however, has very little to do with America, or even New Jersey for that…
Just days before Christmas 2016, the FDA gave a present to some of the children in the United States—they approved the first treatment ever to treat spinal muscular atrophy (SMA).…
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