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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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Idiopathic pulmonary fibrosis

The Continuing Evolution of Our Understanding of IPF

One of the most important aspects of the scientific process is that assumptions are tested, and discarded when discredited, and new hypotheses take their place, with the same stringent measures firmly in place to ensure that they are rigorously tested as well. In other words, medicine is an evolving discipline; going a step further, it…


Prescribing Physical Exercise to Improve Outcomes in PAH

Throughout most of human history, people did not relate exercise with health to the same degree that we see today. This was probably because people were already getting ample amounts of physical activity without the need for additional prompts. It was not more exercise that people needed; it was less of it.  Fast forward to…

factor VIII

Strategies for Managing Inhibitor Development in Hemophilia

The therapeutic use of factor replacement therapy—the mainstay of hemophilia treatment—is impeded by a stubborn problem that can sometimes drastically reduce its efficacy: the development of inhibitors to the coagulation factors administered.  Why does factor replacement therapy go swimmingly well in many patients with hemophilia, yet for some patients, inhibitors develop almost immediately? The short…

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