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Latest Patient Perspectives

Healthy Living With AATD Requires Discipline, Knowledge

There are several things I would have done differently after my alpha1-antitrypsin deficiency (AATD) diagnosis. I did have a great doctor, but there were certain things I wish I had realized were so important. I once heard someone else with AATD say: “Nothing about respiratory illness is straightforward.” While this is true, so much can…

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.

Latest HCP Insights

Cardiovascular system

A Heart-Centered View of Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy characterized by progressive muscle weakness and atrophy. Though most investigations undoubtedly focus on the muscle, increasing evidence suggests that cardiac malfunction is a relevant cause of morbidity and mortality in patients with DMD. "With advanced DMD management and ventilatory support widely applied from 1990s,…

Alagille Syndrome Etiology

Genome Sequencing Increases Diagnostic Accuracy in Alagille Syndrome

In a way, the COVID-19 pandemic has given the world a crash course in basic medical practices and terminology. For example, most of the world now knows how to wear a surgical mask correctly, and medical words such as “quarantine” and “pulse oximetry” have become ingrained in our collective vocabulary.  Another pair of terms that…

Exploring Noninvasive Imaging in Intrahepatic Cholangiocarcinoma

There is an inside joke within the medical fraternity about the enthusiasm that surgeons exude in favor of invasive treatment during multidisciplinary meetings. The characterization is that while invasive treatment is usually seen as a last resort by physicians that belong to other disciplines, surgeons often see it as first-line therapy.  Unfair (or perhaps sometimes…

Meeting Coverage