First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most ... Read more
Overactive bladder is a common problem in men with adrenomyeloneuropathy (AMN), a type of adrenoleukodystrophy (ALD), and also women who are carriers of the disease. What is overactive bladder? Overactive bladder is ... Read more
An infusion of anti-inflammatory mesenchymal stem cells (MSC) given two boys with the cerebral form of adrenoleukodystrophy failed to halt or prevent disease progression, a case study reported. Whether ... Read more
The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. ... Read more
Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see ... Read more
Magenta Therapeutics’ stem cell therapy MGTA-456 leads to clinically meaningful and lasting benefits in neurological and cognitive functions — as well as a resolution in brain inflammation — in ... Read more
The number of treatments for children with rare diseases has grown over the past decade, according to a new study. However, despite the increase, nearly 7,000 rare diseases are still ... Read more
In recognition of Rare Disease Day Feb. 29, BioNews Services launched a social media campaign last month asking patients to describe what makes them rare. Running Feb. 7–29, the ... Read more
Beginning on Feb. 29, Rare Disease Day, chapters from notable scientific books and clinical review articles covering rare disorders will be available free-of-charge from Elsevier. The offer runs through April 30, ... Read more
A new genetically modified earthworm model, Caenorhabditis elegans (C. elegans), mimics the features of human X-linked adrenoleukodystrophy (X-ALD), and could help in more quickly finding targets for therapies and at lower cost, a ... Read more
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