How Shortages Affect the Rare Disease Community

It’s no secret that COVID-19 has caused a lot of issues for not just the United States, but the entire world. Because of the pandemic, for example, we’ve recently been experiencing issues getting the necessary medical supplies for my 4-year-old son, Cayden, who has infantile-onset Pompe disease. Cayden…

Surge in Telehealth Brings Access, Convenience to Rare Disease Patients

A rare disease puts an economic burden on the patients, families, and caregivers that it affects, and will no doubt be an integral part of discussions on Rare Disease Day 2022, which brings international awareness about the more than 300 million people living with rare disorders. Part of that…

Experts Warn Against Interrupting Pompe Treatment During Pandemic

At a recent webinar, experts cautioned that it could be dangerous for people with Pompe disease to stop treatment due to the COVID-19 pandemic. The disease that would come to be called COVID-19 was first reported in the U.S. more than a year ago. As it spread across…