hATTR-PN

The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for…

It is interesting how human emotions can fluctuate like the ebb and flow of the tide. I am usually upbeat and composed, but at the…

Changes in the function of the retina, in combination with nerve damage in the cornea, can be used as early biomarkers of hereditary transthyretin (hATTR)…

Those of us who are caregivers have many of the same underlying goals. But while we all care for our ill loved ones, our…

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and…

I returned to the gym this week to work out. The last time I hit exercise classes and equipment was in 2016, shortly before my…

Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also…

More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the…

As a caregiver, have you ever felt like you’ve been thrown into the deep end and are treading water just to survive? Perhaps this overwhelming…

Patient enrollment is complete for the Phase 3 NEURO-TTRansform trial testing Ionis Pharmaceuticals’ eplontersen in adults with familial amyloid polyneuropathy (FAP). The trial…