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…
3:15 a.m.? Are you kidding? Why am I awake and staring at a digital clock that says it’s 3:15 a.m.? Ever have a night like that? The more you get…
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…
Image source: https://healthyhandyman.com/ On the website ShortBowelSyndrome.com, people living with the syndrome share their personal stories and experiences and give tips, tools, and advice to others living with short bowel syndrome…
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…
Everyone knows lions are the kings of the jungle/mountain/movie studio. They're brave and ferocious! "Lion" not only describes the personality of one fun little boy living with Hunter syndrome (MPS…
“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…
When you’re a preteen, fitting in (or at least, not standing out) can be a big part of life. Sometimes, you’re just trying to get along with schoolmates, teachers, and…
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…
In June 2017, a film was released showcasing the story of a young girl named Kennedy Hansen who had Batten disease—a disease that causes a damaging buildup of lipofuscins in…
As a kid, I always had a pretty good hold on life. Despite my father being deployed during my formative years, I played sports, made good grades, and had great…
You may have heard someone say: “You don’t look sick.” Few phrases can be as disheartening to someone battling a lifelong, or chronic, illness. Blood diseases are just one such…
The spinal cord is a major part of the central nervous system, and when it becomes inflamed, transverse myelitis results. It often causes a band-like sensation across the trunk of…
One of the first steps toward fixing any sort of problem is identifying the cause. This is true of nearly any problem. This is true of the crack in the…
Feeling alone in your struggles is almost an inevitable side effect of living with a rare disease. You’re unlikely to have a support group in a very close vicinity and…
Allergan and Paratek Pharmaceuticals Inc. recently tested a drug called sarecycline for treatment of severe acne. The medicine is a tetracycline-derived antibiotic that is taken orally, and the best part…
Status epilepticus is a rare and sometimes fatal condition. About 60 percent of those with this super-refractory status epilepticus (SRSE) die, making it the most dangerous type of seizure. This…
Long have these cold-blooded, no-limbed reptiles bore a bad reputation; from the Bible to Disney to Indiana Jones. But are things about to change? According to a new French study…
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