As reported in Nature (an international medical publication), research scientists have uncovered the atomic makeup of an important protein found in those with Alzheimer's Disease. This is being touted as a…
Regular readers of Patient Worthy know the cost of drugs is a huge barrier to treatment for many patients and families. Additionally, the fewer people diagnosed with a condition, the…
WHEW! That week was short, wasn't it? We hope you had an awesome Independence Day Weekend in the US, and Canada Day in our neighbors to the north. This week,…
I was fortunate to have had optimistic role models during my formative years. They taught me that glass wasn't either half empty or half full, because the glass can be…
Has your healthcare provider ever talked to you about using an FDA-approved drug for an unapproved use (sometimes called an “off-label” use) to treat your disease or medical condition? It…
Have you ever tried to fit a square peg into a round hole? Short of taking a mallet to the peg, you’re not going to be successful in pushing it…
I was once walking through an outdoor festival near my hometown. There was a band playing off in the distance, but I didn’t know them or the song they were…
In a surprising development, Canadian researchers have discovered that a common drug used to treat acne may be of benefit to thousands of patients around the world who’ve been newly…
Adrenoleukodystrophy (ALD) is a terrible disease - but tell that to Ethan Jeffcoat, who spent his latest birthday playing with a python, alligator and more! ALD is a brain disorder that destroys…
Nope, we're not joking! US SWAT teams (Special Weapons And Tactics, military weapons-equipped law enforcement teams) have been asked to step in to help with backlogged FDA applications to get experimental treatments designated as orphan…
If you think this summer is hot, last summer was on fire for amyloidosis advancements and support. Our partner, The Amyloidosis Foundation produces a seasonal newsletter for all those interested…
According to the Indiana Lawyer, the case of Mikel An Krueger and her young son is a peculiar and sad one. It starts with the birth of Krueger’s son, AG,…
Imagine living every day with fear that paralysis may be coming. The idea always lingering in the background, ominous. That's the case for Kam Redlawsk. But she does not let…
Oh the joys of a 17-month-old toddler… I remember some of the typical behaviors of those days—the throwing heaving of everything and anything, the opening of the fridge a million…
According to the Daily Mail, Harley Bond is a 4-year-old living in the UK. He has MPS III, or Sanfilippo Syndrome, a rare disease which has no treatment and no…
Maybe you’re someone like me who didn’t know what a Hemophilia Treatment Center is or what it does. I never thought about treatment centers of any type, of course, there…
Losing a child is a loss like no other—the most devastating experience a parent can face. And for one daddy who lost his little girl to mucopolysaccharidosis, a very rare genetic…
“Idiopathic Pulmonary Fibrosis: Partnering with Your Doctor,” is an interactive program for people diagnosed with idiopathic pulmonary fibrosis (IPF) and their caregivers. And you know what's even more awesome about…
Kelley Fox is 23 years old, but developmentally she’s only around 5 or 6 years old. She lost half of her brain due to a disorder called tuberous sclerosis. The…
I'll admit, I don't have sickle cell anemia, but I do have a bad habit: I don't ask for help. No matter the situation—maybe my car has been towed, or…
Kids with spinal muscular atrophy (SMA) have a hard time with an evaluation method known as "pair-matching tests." They have a harder time identifying what number, letter, color, or object…
“Catching some Z’s.” “Crashing.” “Hitting the sack.” “Getting some shut eye.” “Zonking out.” “Getting forty winks.” We have a lot of idioms for sleep. On average, we spend one-third of our…
I first became aware of the bleeding disorder hemophilia when I was in high school reading about Nicholas II, the last Russian tsar. His son had severe hemophilia B, which…
Not only does Pamela Looney have hypereosinophilic syndrome (HES), she also has asthma and has suffered from a tumor. At only 15, the sudden development of this tumor severely affected…
Rare disease research usually isn’t the first thing med students think to specialize in. Mostly, because it’s such a small field and it’s not extensively covered during medical school. Let's…
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