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…
The first US patient has been dosed with the drug MIN-102 as part of a clinical trial into its effects in patients with adrenomyeloneuropathy. For more information about this, you…
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,…
A Phase 1/2 clinical trial of the experimental drug NV1205 in patients with childhood cerebral adrenoleukodystrophy (CCALD) has been initiated, announced NeuroVia Inc. The source article can be found here,…
Is there a genetic condition in your family? Are you concerned about the potential for passing this on to your children one day? Do you know if you are a…
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…
Patient Worthy recently attended ALD Life’s Community Weekend 2018, which brought together people and families with Adrenoleukodystrophy (ALD) and Adrenomyeloneuropathy (AMN), and scientists, researchers, and doctors. One of the speakers…
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…
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…
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