U.S. Food and Drug Administration

FDA Places Clinical Hold on FORTIS AT845 Gene Therapy Trial

The U.S. Food and Drug Administration (FDA) has placed a clinical hold on the FORTIS Phase 1/2 trial of AT845, a single-dose gene therapy for adults with late-onset Pompe disease (LOPD). The decision was due to a study participant developing peripheral sensory neuropathy — nerve damage outside the brain…

We’re Eyeing a Medication Switch to Nexviazyme

Recently, my 4-year-old son, Cayden, had an appointment with his metabolic specialist. This doctor manages everything related to his Pompe disease and is part of a team of doctors that diagnosed him when he was just 1 month old. They also oversee everything related to his enzyme replacement infusions.

FDA Efforts to Bring Patients into Treatment Decisions Focus of NORD Webinar

The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…

HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.