Disparities diagnosing rare genetic diseases in minority racial communities likely are due to systemic barriers, and are not because genetic…
Articles by Marisa Wexler, MS
Running clinical trials for any rare disease is difficult, but steps like using innovative trial designs and taking advantage of…
Since its launch 12 years ago, a program to incentivize drug development for rare diseases that affect children has led…
A novel diagnostic algorithm used in a pilot study in Germany was able to provide diagnoses for hundreds of rare…
Scientists have created a novel tool called SynthMD that can be used to generate artificial datasets for rare diseases,…
People living with rare diseases frequently end up being responsible for communication between their healthcare providers, a new study from…
The U.S. Food and Drug Administration (FDA) has agreed to review an application seeking the approval of Upstaza (eladocagene…
Mississippi Gov. Tate Reeves has signed into law a bill that establishes a rare disease advisory council (RDAC) in the…
The World Orphan Drug Alliance, known as WODA — a global coalition of pharmaceutical partners with rare disease portfolios…
The U.S. Food and Drug Administration (FDA) has responded favorably, in recent years, to the use of real-world data in…