hATTR-PN

A woman with familial amyloid polyneuropathy (FAP) developed a type of kidney disease called focal segmental glomerulosclerosis — characterized by scarring in the kidneys — several months after starting treatment with Tegsedi (inotersen), according to a recent case report. Once the patient stopped taking Tegsedi, the kidney…

It’s been over four months since I lost my husband, Aubrey, to hereditary ATTR amyloidosis. The vacuum he’s left behind in the lives of our four children and me may not be immediately apparent, but one only needs to dig a little deeper to see how much we…

Soleo Health, a U.S. national provider of pharmacy services, announced that Amvuttra (vutrisiran), an approved treatment for adults with familial amyloid polyneuropathy (FAP), has been added to its portfolio of specialty treatments. Amvuttra was developed by Alnylam Pharmaceuticals to treat FAP, also known as hereditary transthyretin-mediated (hATTR) amyloidosis with…

Life is full of twists and turns. I was a caregiver for nine years until this past May, when my husband passed away from hereditary ATTR amyloidosis. Aubrey was diagnosed in 2013, and despite the many challenges we both faced, we never once thought to give up. Unfortunately,…

Treatment with Tegsedi (inotersen) for more than three years continued to slow disease progression and improve the quality of life in people with familial amyloid polyneuropathy (FAP), according to new data from the extension study of the NEURO-TTR trial. Patients who started treatment earlier experienced greater benefits, but…

The European Commission has approved Amvuttra (vutrisiran) as a treatment for familial amyloid polyneuropathy (FAP) — also known as hereditary transthyretin-mediated (hATTR) amyloidosis with polyneuropathy — for people in the EU with stage 1 or stage 2 polyneuropathy. The decision was announced by the medication’s developer, Alnylam…

I recently attended an amyloidosis scientific conference in Heidelberg, Germany, that was a productive and empowering experience. As a former caregiver to a spouse with hereditary ATTR amyloidosis and a patient advocate with New Zealand Amyloidosis Patients Association, learning about the advancements in the field of disease…

Onpattro (patisiran), a widely approved treatment for familial amyloid polyneuropathy (FAP), preserved functional ability and improved life quality for people with a related form of heart disease called transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy. That’s according to top-line results from the Phase 3 APOLLO-B clinical trial, which tested the…

Two weeks after the passing of my husband, Aubrey, my four children decided that the company of a new puppy would be the best way to cheer me up. Looking back now, almost four months after Aubrey left us on May 22, Akira — an…

Eplontersen reduced transthyretin (TTR) protein levels, eased disease progression, and improved quality of life in adults with familial amyloid polyneuropathy (FAP), according to an eight-month interim analysis of the NEURO-TTRansform study. Based on these data, the investigational therapy’s co-developers Ionis Pharmaceuticals and AstraZeneca will seek regulatory approval…