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Browse Rare Disease Advisor’s engaging “Rare Care” podcast series, featuring interviews with leading researchers, stakeholders, and influencers within the rare disease community.

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How Can Adherence to Imatinib Therapy in Patients With GIST Be Improved?

An insidious problem facing clinicians worldwide is patient nonadherence to the treatment regimen prescribed. In these situations, even diseases that have curative potential are at risk of further deterioration if patients refuse to take their medications as prescribed.  Scientists are now realizing just how big of an issue nonadherence is. There are a few hypotheses…


Paroxysmal Nocturnal Hemoglobinuria: The Great Impersonator

Paroxysmal nocturnal hemoglobinuria (PNH) is characterized by chronic intravascular hemolysis, which is brought about by the continual activation of alternative pathways of the complement system—an integral part of the innate immune system. This disease is notoriously invasive, and the rate of correct diagnosis upon first encounter is low. Nevertheless, the past few years have seen…

Human heart anatomy

Treating Cardiac ATTR: Current Strategies and Challenges

For many years, the treatment of cardiac hereditary transthyretin amyloidosis (hATTR) relied on liver transplantation to remove the source of aberrant transthyretin (TTR). However, several studies have shown that liver transplantation provided an incomplete cure. Hence, combined liver/heart transplantation has emerged as an alternative. In addition to obvious transplant-associated limitations, such as the dependence on…


Life Aspirations Explored in Patients With DMD

It is unfortunate that the prevailing view on chronic illness is that it simply “happens” to someone, and that the best one can do is to cope with the difficulties attached, comply with treatment, and hope for the best.  This is perhaps exacerbated by medical language; a patient is said to have been “diagnosed with”…

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