In an twist of fate, a brand new gene therapy for the treatment of Adrenoleukodystrophy (ALD) uses one of medicine's greatest nemesis, HIV, to treat this fatal genetic brain disease-- and…
Adrenoleukodystrophy is a rare hereditary condition which affects the myelin sheath and the ability of the body to process a type of fatty acids. The most damaging form primarily affects…
Minoryx Therapeutics is on their way to making strides for patients with adrenomyeloneuropathy, also known as AMN, reports Labiotech. They just started their phase II initiative to test patients for…
The memories we have of growing up should be the type that we look back on and smile about. That is not to say that they all should be happy…
Persistence is a tricky thing. In Hollywood films, persistence pays off. The dorky kid keeps asking the cheerleader out while he’s helping her with her homework until she finally agrees…
Sometimes it takes a tragedy to create real change. Last year, Connecticut joined the short list of states to implement an ALD newborn screening test, after the parents of a…
While cliché, it is still true that children are the future. That is why it is so important that we find ways to protect them where we can and prepare…
Aidan Jack Seeger was a curly-headed, blonde boy with incredible blue eyes. At age seven he succumbed to a rare disease called adrenoleukodystrophy (ALD)—only 11 months after his diagnosis. Had…
Adrenoleukodystrophy (ALD) is a genetic disorder that is very rare and usually occurs in childhood. People with ALD have two options for treatment, both of them less than favorable and…
If you’re anything like me, you probably don’t remember much about the work you did 20 years ago. And unless you’re a pack rat, chances are you can’t even lay…
Jon Milne is on a quest to race across the seven seas to help raise awareness of rare disease! Jon has always been a keen sportsman, aged 30 he had…
Happy August Patient Worthy Peeps! This week, we have some awesome posts to keep you up-to-date on some of the latest rare disease developments. There's news on a hopeful possible…
There’s a lot happening in the adrenoleukodystrophy (ALD) world, so here’s the latest scoop. Adrenoleukodystrophy, or ALD for short, is a genetic rare disease that destroys the protective sheath surrounding…
NeuroVia, Inc. is a rare disease pharmaceutical company that primarily focuses on finding treatments to rare neurological diseases. Markedly, this private company is especially concerned about the effects of the…
The United Leukodystrophy Foundation is hosting two two-day long events for families and medical professionals! Here's all you need to know about it before registering: What: Cocktail reception and dinner, fascinating…
In writing for Patient Worthy, we’re privileged to discover some amazing stories of how people live with—and live through—the adversity of having a rare disease such as adrenoleukodystrophy (ALD). Sadly,…
I recently got together with a sweet friend, who has a six-month-old. It was the first time we'd actually talked face-to-face since her daughter was born, and the first time…
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…
Second grade. So big but so little. An academic year filled with spelling and vocabulary words, simple math fast facts, and learning how to use a keyboard. And maybe at…
A firefighter, also known as a fireman, is a highly skilled man or woman who works to combat and extinguish fires. They also take steps to prevent fires, act as…
The word adrenoleukodystrophy doesn't exactly roll off the tongue, does it? Adrenoleukodystrophy (ALD) is an exceptionally rare genetic disease that affects one in 18,000 males. If you're one of those 18,000 people,…
This week is a small win for adrenoleukodystrophy (ALD) awareness and for these mini-bikers. Kevin Bean're and Tom Ford are mini-biking across the country, from Florida to California, to raise awareness…
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