Twenty genetic therapies have been approved by the US Food and Drug Administration to
date, a number that now includes the first CRISPR genome-editing therapy for sickle cell …

Background Over the past decades, US Congress enabled the US Food and Drug
Administration (FDA) to facilitate and expedite drug development for serious conditions …

Drug repurposing—identifying new therapeutic uses for approved drugs—is often a
serendipitous and opportunistic endeavour to expand the use of drugs for new diseases …

Of the several thousand diseases that affect humans, only about 500 have treatments
approved by the US Food and Drug Administration. Even for those with approved …

Highlights•FDA approved 176 new drugs for the 20 leading pharmaceutical companies
(2014–2023).•An additional 33 new drugs were acquired through post-approval acquisition …

Despite general advancements in population health indicators and universal health
coverage, people living with rare diseases and their families still experience considerable …

Little is known about patients' and families' lived experiences of participating in pediatric
gene therapy (GT) clinical trials. Currently, pediatric GT research targets a broad range of …

The introduction of safe and effective rare/ultra‐rare disease treatments is a focus of many
biotherapeutic enterprises. Despite this increased activity, a significant unmet need remains …

Over the past 4 decadessince the Orphan Drug Act was passed in 1983, there have been
tremendous advances in research, innovation, and regulatory policies that have spurred …

Background Insufficient patient enrollment per month (= accrual) is the leading cause of
cancer trial termination. Objective To identify and quantify factors associated with patient …