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…
News
Onpattro (patisiran) is more effective than Tegsedi (inotersen) at reducing neurological impairment and improving quality of…
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…
HATTR-PN
Health InsightsFirst ATTR Patient Dosed in Phase 1 Trial of Intellia’s Gene-editing Therapy NTLA-2001
The first patient has been dosed in a Phase 1 clinical trial evaluating NTLA-2001, Intellia Therapeutics’ investigational gene-editing therapy for the treatment…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of free virtual workshops in December,…
Eidos Therapeutics announced that the Phase 3 clinical trial evaluating its lead candidate acoramidis (formerly known as AG10) in treating adults with transthyretin amyloidosis cardiomyopathy (ATTR-CM)…
Although less able to form damaging protein deposits, the E61K variant of transthyretin appears to cause familial amyloid polyneuropathy (FAP) through other mechanisms,…
A photo of a bespectacled young boy, his red baseball cap slightly askew as he enjoys time outside, will be featured on the front cover…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like the option of continuing its…
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