People living with rare diseases frequently end up being responsible for communication between their healthcare providers, a new study from…
Articles by Marisa Wexler, MS
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…
Genetic testing done for a 55-year-old woman diagnosed with an unusually mild case of AADC deficiency revealed a disease-causing…
Treatment with sertraline, an antidepressant sold under the brand name Zoloft among others, worked to ease anxiety in a young…
The first successes of gene therapy for AADC deficiency highlight the potential that gene therapies have to improve life…
Following gene therapy, people with AADC deficiency should receive a few weeks of intensive physical therapy, with additional physical…
Researchers say childhood epilepsy and mild attention deficits were effectively “hiding” a case of mild aromatic l-amino acid decarboxylase…