Through its recently issued press release, bluebird bio announced the FDA’s accelerated approval of the first therapy to slow the progression of adrenoleukodystrophy (ALD). The disease is the result…
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…
Have you ever heard of Orphan Drug designation before? The FDA grants Orphan Drug designation to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. In America,…
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…
bluebird bio has created a gene therapy for the treatment of cerebral adrenoleukodystrophy (CALD), which is now making its way through a clinical development program. According to Businesswire, bluebird has…
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…
In November 2020, Viking Therapeutics ("Viking") shared insight into their metabolic disease program. According to Greg Zante, the Senior VP (SVP) of Finance for Viking, the company's treatment VK2809, licensed…
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…
When this 20-year-old male patient was serving in the military, he came down with two severe medical issues: pneumonia and adrenal crisis, categorized by an issue with an adrenal hormone…
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 an article in BioPortfolio, Magenta Therapeutics, a biotechnology company based in Cambridge, Mass., recently announced updates to its Phase II trial of MGTA-456 for the treatment of…
A new precision medicine center, specifically for pediatrics, has launched in Utah. This new center is the result of collaborative efforts from the Intermountain Primary Children's Hospital, Intermountain Precision Genomics,…
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…
According to a story from PharmaTimes, the gene therapy company Bluebird bio has released updated results from a clinical study of its Lenti-D gene therapy as a treatment for patients…
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 Ysios Capital, the biotechnology company Minoryx Therapeutics recently announced that the first patient has been dosed in the company's phase 2 clinical trial. This trial…
Friedreich's Ataxia Friedreich's ataxia (FRDA) is a rare disease which is caused by a frataxin deficiency. This deficiency results in the degeneration of nerves within the spinal cord. This nerve…
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…
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…
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