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Rare Care Podcast

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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Human microbiota

Is Gut Microbiota Involved in Pulmonary Arterial Hypertension?

Pulmonary arterial hypertension (PAH) is primarily associated with abnormal changes to the pulmonary vasculature, which eventually leads to irreversible right heart failure. Pathologists characterize endothelial dysfunction and pulmonary arterial fibrosis as the main drivers of vascular stiffening.  Over the last few years, scientists have painstakingly put together a picture of the network of pathological processes that…

Red blood cells

The Dangers of Delayed Diagnosis in Acquired Hemophilia A

Acquired hemophilia A differs from classical hemophilia A in that it occurs in individuals without a previous history of bleeding. Both diseases involve neutralizing autoantibodies acting against coagulation factor VIII (FVIII). However, in acquired hemophilia A, patients typically present with an isolated prolonged activated partial thromboplastin time (aPTT) that is later discovered to be due…

B cells

Existing Immunosuppressive Strategies for Treating Myasthenia Gravis

Myasthenia gravis is a neuromuscular disorder driven by autoimmunity against the postsynaptic neuromuscular junction of skeletal muscles. The clinical manifestations of myasthenia gravis are broad, but generally it can be characterized as being fluctuating, chronic, and oftentimes carrying a poor prognosis.  “[Myasthenia gravis] impairs [neuromuscular junction] transmission and can manifest in a spectrum ranging from…

MTC complications

Treating Advanced Medullary Thyroid Carcinoma

The history of medicine, stretching as far back as when our human ancestors first roamed the earth, is a poignant volume of how far we have come, and how far we have yet to go.  Take cancer, for example. In the past, when life expectancy was drastically lower, cancer was seen as less of an…

cystic fibrosis medical devices

The Evolving Face of Cystic Fibrosis Care

I distinctly remember whispers when I was in medical school about potentially groundbreaking treatments in the works that might finally make cystic fibrosis an illness of the past. Regrettably, these predictions have not manifested; cystic fibrosis is still very much a condition that carries a high morbidity and mortality rate.  It is tempting to desire…

experimental CCA therapies

Targeting DNA Damage Response as a Therapeutic Strategy in CCA

Cholangiocarcinoma (CCA) is an aggressive type of cancer with limited therapeutic options available. When possible, patients are offered surgical resection, whereas unresectable cases are managed with adjuvant treatment. Chemotherapy or radiotherapy are usually used to increase the chances of recovery and improve patient's quality of life and survival. However, despite the recent advances, the survival…

Meeting Coverage