Patient Worthy partner Alex TLC has announced that, in a large-scale newborn screening study supported by the UK's NHS and Genomics England, adrenoleukodystrophy (ALD) and metachromatic leukodystrophy (MLD) will be…
According to a story from Greenock Telegraph, Michael Conway of Port Glasgow, UK lives with adrenoleukodystrophy, a rare disease. Recently he received a letter of support from William and Kate,…
When Chantel and Jason Funk learned that their son Bowin has adrenoleukodystrophy (ALD) in 2019, they thought that the questions about the future, and how to manage the condition,…
According to a story from wmur.com, a 12-year-old boy named Grady Smith from Salem, NH lives with adrenoleukodystrophy, a rare disease. Grady is a big fan of basketball (his favorite…
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Growing up, Priscilla Veneklause watched as her father struggled to manage his rare genetic disorder: adrenoleukodystrophy (ALD). Unfortunately, her father lost his fight with ALD when Priscilla was still…
In May 2016, I was diagnosed with a rare genetic neurological disease called adrenoleukodystrophy (ALD). Sadly, it's incurable and there's no medication to ease my condition. Since then, my life…
According to a story from the Glasgow Times, David Steed of Paisley, UK, was diagnosed with adrenoleukodystrophy when he was 18 years old. At the time, he didn't have any…
According to a press release, the gene therapy company bluebird bio, inc. has recently announced that its gene therapy elivaldogene autotemcel (marketed as SKYSONA) has received Accelerated Approval status from…
In May 2016, I was diagnosed with a rare genetic neurological disease called adrenoleukodystrophy (ALD). Sadly, it's incurable and there's no medication to ease my condition. Since then, my life…
Science Magazine recently joined other news sources in reporting that Bluebird Bio’s Phase 3 clinical trial investigating a treatment for a neurological disease was put on hold by…
Michael Conway, a grandfather from Scotland, believes that there should be increased rare disease screening after he was diagnosed with adrenoleukodystrophy (ALD) in 2016. Like many other rare disease patients,…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
A rare disease diagnosis is often shocking, and it means making life changes. An adrenoleukodystrophy (ALD) diagnosis is no exception. Because this condition typically impacts children, parents have to make…
Living with a rare disease during this pandemic is scary. There are additional concerns, risks, and worries that must be considered. Those affected by adrenoleukodystrophy have to keep many of…
According to a story from pmlive.com, the gene therapy company Bluebird Bio's latest recently released results from the company's phase 2/3 clinical trial demonstrates further encouraging data for its Lenti-D…
In May 2016, I was diagnosed with a rare genetic neurological disease called adrenoleukodystrophy (ALD). Sadly, it's incurable and there's no medication to ease my condition. Since then, my life…
According to a study published in the Orphanet Journal of Rare Diseases, a study that was intended to identify a new approach to monitoring the progression of x-linked adrenoleukodystrophy in women…
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…
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…
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…
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…
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