Recent news headlines have sparked significant concern among those of us in the pulmonary hypertension (PH) and other rare disease communities. The massive budget and staff cuts across multiple federal agencies are particularly troubling, prompting a flurry of conversations both online and in private. As we navigate these challenging…

I think it is more effective to watch for good advice instead of asking for it. An Inc. article titled “25 Excellent Pieces of Advice That Most People Ignore” covers words of wisdom that often go in one ear and out the other. “It’s easy to find someone to…

Note: This story was updated July 13, 2022, to correct the name of Rare-X’s CEO Charlene Son Rigby. Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world.

The European Commission is expected to propose a new governing framework for health data next month, called the European Health Data Space (EHDS), with the aim of connecting national health systems to facilitate secure and efficient transfer of data across systems in different European nations. The move is expected to…

Patient advocacy is a broad term covering many topics, and the concept leaves many confused. Those of us in the pulmonary hypertension (PH) community must educate ourselves about it. In this column, I primarily refer to self-advocacy and briefly touch on systems advocacy. To me, self-advocacy means making my…

As anyone affected by a rare disease knows, treating the illness while trying to go about everyday life is an expensive undertaking.  But exactly how expensive — in terms of direct and indirect costs across rare disease populations — might still come as a surprise: almost…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

First in a series. Those in the pulmonary hypertension (PH) community often refer to each other as PHamily. They don’t live under the same roof, but they do have a strong understanding of what life is like for other patients and caregivers: endless doctor appointments, hospitalizations, procedures, and surgeries. Their…