For now, Ann Marie Harte’s five-year-old son, Lewis, can still walk. He can no longer run, and he has a hard time with the stairs. The trampoline exhausts him, and…
Lawyers in Texas were enraged. They demanded the government give a 10-year-old girl with a rare disease back to her family. Rosa Maria Hernandez is a 10-year-old girl with cerebral…
At some point in your life, you might have taken a melatonin supplement from a bottle to get to sleep, or to get over jet-lag. Your body naturally produces melatonin…
Good news for mantle cell lymphoma patients! A new treatment called Calquence has just received approval from the U.S. Food and Drug Administration (FDA) for adults who suffer from this rare…
Rare disease research can be daunting. It's much more difficult for rare, or orphan, diseases to find adequate funding to develop treatments than it is for diseases that have larger…
If you or someone you love have Friedreich's Ataxia, you know how badly we need an adequate treatment. Friedreich's Ataxia (FA) is a rare disorder, which results in neurological and…
Happy Friday! We have some great news for the short bowel syndrome community. We also have info on a PBS doc you won't want to miss. One of our contributors…
Up until now, diagnosing ovarian cancer hasn't been so easy. Most of the time, it goes undiagnosed until it's already advanced. At that point, treatment is harder and less reliable.…
Dogs, with their pure hearts and boundless supply of affection, have made life better for humans for a long time. Dogs aren't just making people feel better by giving them…
Elliott Cunnew, from Collaroy, Australia is what you might call a cool dad. Elliot is the father two kids, 20-year-old Amelia, and 17-year-old Lachie. During the day, he sells private…
Painters, photographers, sculptors, and visual artists that defy categorization, take note! The EveryLife Foundation for Rare Diseases' Rare Artist Contest is back, for the ninth year in a row. The…
I love Thanksgiving! It's a great time to catch up with friends and family. The one drawback is my diet is strictly controlled due to my rare disease, so at…
A perfectly coifed woman-child cuts you off on the way to the YMCA. Your children are copping some major attitude. Your spouse forgets to grab milk for dinner. Do you…
If you or someone you love has Cushing Disease (often confused with the umbrella term, Cushing Syndrome), you've probably heard about microadenas. Microadenas are the tiny, benign tumors that develop…
It's not every day that you hear about how nicotine may help lung disease. However, researchers at The Ohio State Wexner Medical Center are asking this question. They're testing whether…
Happy Pre-Halloween Patient Worthians! October is Dwarfism Awareness Month. Not only that, MDS World Awareness Day was just two days ago! Get the deets below. We also have a story…
Earlier this month, PBS released the "The Gene Doctors." The hour-long documentary follows the recent emergence of gene therapy as a novel method of treating rare diseases. The film discusses…
Vegan, gluten-free, dairy-free. Three symbols and phrases I constantly look for in food. I have to. Having chronic illness and rare disease has a tremendous impact on my digestion. Not…
This past Sunday, October 22nd, Steve Nugent, 53, crossed the finish line at the 42nd annual Marine Corps Marathon in Arlington, Virginia. A big group of the people who loved him…
You may have heard the buzz during the Zika outbreak in 2016. Many professionals suspected a relationship between the Zika virus and Guillain Barre Syndrome (GBS). Guillain Barre Syndrome is…
Check out part 1 of this LGS post here. After prepping: Arrive at the airport early – Ask the airline agent at check-in about getting your wheelchair assistance. Use this…
Spinal muscular atrophy (SMA) is the leading genetic cause of infant mortality in the USA. So why aren't hospitals in Texas screening newborns for it? That's what a group of…
Traveling is certainly possible and can be more relaxing with some pre-planning and organization. Some of these ideas may not be applicable to you and your family. I have compiled…
There's a new treatment for X-Linked Hypophosphatemia (XLH) that may be available in the not-so-distant future. XLH occurs when the kidneys process phosphate abnormally, leading to excess phosphate loss in…
Happy Friday Patient Worthians! This week, we some news to be happy about! We have an awesome story of how thinking like a start up will help lead to more…
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