Knowing you’re not alone and connecting with others who share the same experience with Dravet syndrome can be incredibly valuable. Parenting a child with such a specific set of symptoms…
I know many people who would love to go to a local support group, but there either isn’t one in your area or you are not well enough to attend.…
What can one little boy and his family do to fight a rapidly debilitating and (currently) incurable disease such as Duchenne muscular dystrophy? A lot. The diagnosis of a loved…
We constantly hear about cancer deaths, but every once and a while, we hear the rare cases of cancer survivors. Linda VanDershaaf is one such survivor and today she is…
Having a peanut allergy is nothing to sneeze at. If you are a parent with a child who has a peanut allergy, you need to be constantly vigilant. Now, a…
According to a press release put out by PTC Therapeutics, Inc., the Office of Drug Evaluation of the Food and Drug Administration on Wednesday, Oct. 25th, 2017, sent a complete…
Popular wisdom tells us that the first step to fixing a problem is admitting that you have a problem. This works for individuals, like alcoholics, thanks to William Griffith Wilson,…
Alkaptonuria, a rare genetic metabolic disorder resulting from excess homogentisic acid in the body, affects all kinds of people, though symptoms tend to become more severe in males. Symptoms include:…
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…
The FDA this week granted orphan drug designation to glepaglutide for the treatment of short bowel syndrome (SBS), developed by Zealand Pharma A/S. SBS is a rare disorder where an…
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…
We sometimes hear about iron storage problems, but what does that actually mean? Genetic hemochromatosis (GH) is, as the name clearly states, a genetic disease. That means it's one that…
Breast cancer, BRCA1, and BRCA2 are very much in the news these days. While the BRCA mutation is rare, 1 in 8 U.S. women will develop invasive breast cancer, according…
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…
Our bodies are complex and unpredictable machines. We can try our best to keep them healthy, but sometimes nature does its work, resulting in something we never could have planned…
Too much of a good thing can be dangerous. For people with acromegaly, a rare disease caused by too much growth hormone (GH), an excess of this otherwise helpful growth factor can…
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…
When Kent Allen was told he had a year to live, he was hopeless. A year simply wasn't enough time. According to Waikato Times, over ten years ago, Kent was…
All a Hollywood star needs to do is be seen with a designer outfit, bag, or shoes and that company can be propelled from anonymity into the white, hot spotlight.…
The Food and Drug Administration has approved the second cancer treatment that will be totally personalized, proving that these kinds of treatments will become a regular thing moving forward. The…
Ten-year-old Coleman Walsh always wanted to be a professional hockey player and now his dream has come true. According to the Daily Mail, he just signed with the Massachusettss's Babson…
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…
One young lady is working to bring awareness to Gaucher disease through blogging. Not only does Lauren Edwards share inspirational posts and pictures, she also shares the personal stories of…
What happens when you’re not the cancer that everyone is talking about? Funding, research, and headlines can often be noticeably lacking when you’re just a run-of-the-mill, life-threatening gene mutation, according…
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