hATTR-PN

I look for humor in every situation. Humor can be lifesaving in times of despair. U.S. Congregational minister Henry Ward Beecher said, “A person…

The European Medicines Agency (EMA) has agreed to review Alnylam Pharmaceuticals’ application requesting the approval of vutrisiran, its second-generation RNA interference (RNAi) therapy candidate…

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community…

The 3rd European ATTR Amyloidosis Meeting for Patients and Doctors was held virtually on Sept. 6-8. Because the New Zealand Amyloidosis Patients Association,…

Vutrisiran, an investigational therapy for familial amyloid polyneuropathy (FAP), eased neurological impairments, lessened heart stress, and ameliorated quality of life, nutritional status, and…

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare…

My husband and I were married on March 15, 1995. Looking back at these 26 years, I can see the gray and black stain of…

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which…

The melodic chirping of birds outside the window is louder this morning. This is because Auckland, the city in New Zealand where I live with…

Long-term treatment with Tegsedi (inotersen) helps preserve quality of life in people with familial amyloid polyneuropathy (FAP) for up to three years, according…