hATTR-PN

Onpattro (patisiran) results in sustained improvements in polyneuropathy, quality of life, and other symptoms among people with familial amyloid polyneuropathy (FAP), while showing…

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their…

Onpattro (patisiran) is more effective than Tegsedi (inotersen) at reducing neurological impairment and improving quality of…

I feel the coolness of the glass against my forehead. The shower is pelting warmth against my skin, but the water doesn’t feel very soothing…

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient…

Three recently approved therapies for treating familial amyloid polyneuropathy (FAP), also known as hereditary transthyretin amyloidosis with polyneuropathy, have won their developers Best…

“I love you. If anything happens to me, you know where I keep the account details.” My husband uttered these parting words as I dropped…

Having a chronic progressive disorder such as familial amyloid polyneuropathy (FAP) can be both stressful and isolating.  Recreational therapy is a form of treatment…

The first patient has been dosed in a Phase 1 clinical trial evaluating NTLA-2001, Intellia Therapeutics’ investigational gene-editing therapy for the treatment…

A liver, donated by a patient with familial amyloid polyneuropathy (FAP) caused by a Ser50Arg mutation in the transthyretin (TTR) gene, caused amyloidosis in the…