When Karter was first born 14 months ago, his mother Breonna was overjoyed. But she quickly realized a series of upcoming obstacles when, at birth, Karter was diagnosed with Alagille…
Normally, the standard FDA review process for potential drugs or biologics takes around 10 months to complete. However, when a drug receives Priority Review status, it means that the FDA…
According to a recent press release from Pfizer and biotechnology company Vivet Therapeutics, VTX-801, a gene therapy candidate for patients with Wilson disease, recently received Fast Track designation from the…
When organizations partner together to work towards improving patient outcomes, it can also help to increase the understanding and spur research of certain conditions. According to Healio, a recent…
Normally, Fast Track is an FDA process designed to facilitate and expedite the development and review of drugs or biologics intended to treat rare or serious conditions. Recently, Healio…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
On August 17, 2021, Amnesty International released an urgent call to action to provide protection in Malawi for those with albinism. This call for change came following the death…
Sometimes, it can be difficult to acquire treatment options for patients with rare diseases. Brand name products may also be expensive and relatively inaccessible, prompting the need for generic…
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