The Food and Drug Administration (FDA) on Tuesday granted expanded approval to Wellcovorin for the ultra-rare disease cerebral folate deficiency (CFD) in both children and adults, the same ...

The FDA proposes new rules allowing mutation-specific gene therapies to qualify for approval despite extremely small patient ...

If your child were critically ill, how far would you go to find answers?

This year’s Rare Disease Day event Feb. 27 at the Jacobs School of Medicine and Biomedical Sciences focused on updates in research and diagnostics, and advancements in treatment.

Thus far, 7,600 genetic diseases have been identified and more are being discovered every year. Read more at straitstimes.com ...

A key US Republican senator said he’s launched an investigation into the Food and Drug Administration’s recent denials of ...

Lysosomal Storage Disorders are genetic conditions caused by enzyme deficiencies that lead to harmful substance buildup in cells, resulting in progressive organ damage. Common disorders in India ...

A new federal proposal aimed at speeding approval of gene therapies for ultra-rare diseases could offer renewed hope to North Texans like Kasey and ...

Researchers at VIB and Antwerp University have identified a major genetic risk factor for a rare form of frontotemporal ...

Globally, an estimated 300 million people live with a rare disease. They typically experience isolation, diagnostic difficulties, lack of therapies, fragmented care, stigma, a struggle for recognition ...

The FDA is already under fire from lawmakers for recent delays and rejections of new drugs for rare diseases.