Changes in the nerve fibers of the cornea — the eye’s clear protective outer layer — may help in the…
Articles by Patricia Inácio, PhD
Tegsedi (inotersen) slows the progression of symptoms, particularly muscle weakness and sensation loss, in people with familial amyloid polyneuropathy (FAP), according…
A modified surgical procedure to implant a small filtration device — called Ex-PRESS — was effective at treating glaucoma in most…
Glu89Lys, a rare gene mutation that can cause familial amyloid polyneuropathy (FAP) — and is associated with early onset…
Alnylam Pharmaceuticals will not conduct an interim analysis of its Phase 3 clinical trial testing Amvuttra…
Familial amyloid polyneuropathy (FAP) patients who carry the Val30Met mutation may experience central nervous system involvement early in…
Treatment with Tegsedi (inotersen) for more than three years continued to slow disease progression and improve the quality of…
Larger studies are needed to back the use of nailfold capillaroscopy — a non-invasive technique for imaging tiny blood vessels…
A total of 30 patient advocacy groups working to further understanding and the needs of people living with rare diseases…
Vutrisiran continues to show promise at lessening heart damage in adults with familial amyloid polyneuropathy (FAP) with cardiac involvement, according…