hATTR-PN

Larger studies are needed to back the use of nailfold capillaroscopy — a non-invasive technique for imaging tiny blood vessels in the fingers — for…

Alnylam Pharmaceuticals has selected Orsini Specialty Pharmacy to distribute Amvuttra (vutrisiran), which was recently approved by the U.S. Food and Drug Administration (FDA)…

I recently watched a documentary narrated by British naturalist David Attenborough about how climate change and deforestation negatively affect animal life on our…

A total of 30 patient advocacy groups working to further understanding and the needs of people living with rare diseases have been selected to receive…

“Lots of people want to ride with you in the limo, but what you want is someone who will take the bus with you when…

Members of a Chinese family carrying a rare gene mutation causing familial amyloid polyneuropathy (FAP) experienced abnormal protein deposits in the eye — manifesting…

Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average…

I lost my husband, Aubrey, on May 22. This period of adapting to him being gone has been somewhat challenging for me. Although I…

A one-time treatment with NTLA-2001, Intellia Therapeutics’ experimental gene-editing therapy for people with familial amyloid polyneuropathy (FAP) and other types of ATTR amyloidosis, continued…

If you are new to my column, my name is Jaime, and I was a caregiver to my husband, Aubrey, until he passed…