Whose ready for some Monday motivation to get your week started? Readers of the Wall Street Journal might be familiar with their "What's Your Workout?" series, profiling someone's fitness and…
This is Part 2 of Alexander's Story, click here to read Part 1. During Alex’s journey Jhoanny had gotten some information from NORD’s website and thought about getting involved. After…
Jhoanny’s son Alexander Matthew was born perfectly healthy in 2008. When Alexander was three, Jhoanny noticed he seemed to be drinking a lot of water and sweating a lot. Since…
Rare disease research is often underrepresented in the field of science. However, recent developments have made researchers more interested in studying this group of diseases. For instance, investments in gene…
Erika Baker and her family have been dealing with the impact of adrenoleukodystrophy (ALD) for generations. The disease has impacted several of her family members, including her uncle, her cousins,…
I met Jon at the 2018 United Leukodystrophy Foundation Conference and had the privilege of learning his and his family's story. Jon was born in Wisconsin in 1992, joining the…
In 2001, when I was three years old, my dad was diagnosed with Adrenoleukodystrophy (ALD). ALD is a rare genetic neurological disorder with no effective treatment or cure. Over the…
Lawmakers in the U.S. state of North Carolina have expanded the state’s newborn screening testing to include three more conditions. These are Pompe disease, Mucopolysaccharidosis type 1 (MPS 1), and…
The U.S. Food and Drug Administration has granted Breakthrough Therapy designation to the experimental drug Lenti-D, which is being produced by the gene therapy company Bluebird bio to treat cerebral…
A drug called Anavex 2-73 being developed to treat Parkinson’s disease dementia is planned to undergo testing in a Phase 2 clinical trial, reports Globe Newswire. Parkinson’s disease is a…
Michael Conway, 51, was diagnosed with Adrenoleukodystrophy (ALD) two years ago. Since then, he has been raising awareness for the rare and potentially life-shortening condition. He wants to let people…
A new treatment under development has produced promising results in patients with blood cancers and is now being tested in inherited metabolic disorders, reports Financial Buzz. Inherited metabolic disorders are…
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…
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…
The heart-breaking story of baby Charlie Gard, who recently died of a rare genetic condition called encephalomyopathic mitochondrial DNA depletion syndrome, touched the lives of families around the world. Although…
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…
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…
Don't miss out on two days worth of presentations, roundtable discussions, live and silent auctions, networking and more with the ALD community! Learn about the latest ALD news and get…
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,…
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