Happy Father's Day Week Patient Worthians! We hope you celebrated your rare dads this week-- because they deserve it. As you know, it's Myasthenia Gravis Awareness Month so we are highlighting some new drug…
There's good news for people living with carnitine palmitoyltransferase (CPT) deficiency and their caregivers! Ultragenyx recently released data from an open-label study of the treatment triheptanoin. In the study, five infants…
A year ago, Novartis let people know that its drug, secukinumab, had shown positive results in a Phase III study for the treatment of ankylosing spondylitis. Now, the same drug could…
So the more I read about Lyme disease, the more worried I get. With over 300,000 cases alone reported annually to the CDC, we don’t know how many undiagnosed people…
PW Contributor Alexis Plofchan is 22 years old and a student at William and Mary. She and her parents have been fighting Lyme disease for the past eight years. In…
This is the last segment of Mariana's medical journey written by Mariana's mother, Carolina. Click here for part one and here for part two. Before the three month check up…
A mother of three in Scotland had experienced dystonia symptoms since childhood, and these symptoms got progressively worse over the years. Like so many with a rare condition it took…
The right doctor and the right hospital can make all the difference when your child is diagnosed with a rare (and frankly terrifying) medical condition. That’s what London couple Sukhi…
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