hATTR-PN

This is my 48th column since I began this providential opportunity as a writer at Amyloidosis News Today. To be given the platform to…

An annual decline in sensory nerve function — one greater than 25% — occurs as long as two years before the onset of familial…

Life experience plays an integral role in shaping our behavior and thinking. I have come a long way from being an unsettled 18-year-old to a…

The benefits of Vutrisiran in reducing neurologic impairment and overall disability, and improving the quality of life in adults with familial amyloid polyneuropathy…

New Zealand was largely able to avoid the effects of the COVID-19 pandemic, until now. The contagious nature of the delta variant and the high…

A retrospective study by the National Institutes of Health (NIH) suggests that healthcare costs for those with rare diseases have been underestimated, possibly being three…

My husband, Aubrey, has begun watching cooking videos on YouTube to overcome an aversion to food and eating. We are both from Malaysia, where f…

Note: This story was updated Nov. 24, 2021 to clarify that NTLA-2001 works by disrupting the TTR gene to reduce transthyretin levels.   The U.S. Food…

Global Genes has partnered with the Rare Disease Diversity Coalition (RDDC) to advance health equity for rare disease patients and caregivers in underrepresented…

As we enter the second half of October, I’m not quite sure how the end of the year has arrived so quickly. Time flies while…