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View article: Intracranial calcification in Fam20c-deficient mice recapitulates human Raine syndrome
Intracranial calcification in Fam20c-deficient mice recapitulates human Raine syndrome Open
View article: Long-term efficacy and safety of inotersen in patients with hereditary transthyretin amyloid polyneuropathy with or without cardiomyopathy: post hoc analysis of NEURO-TTR open-label extension
Long-term efficacy and safety of inotersen in patients with hereditary transthyretin amyloid polyneuropathy with or without cardiomyopathy: post hoc analysis of NEURO-TTR open-label extension Open
Background/Introduction Cardiomyopathy (CM) with associated heart failure and polyneuropathy (PN) are common manifestations of hereditary transthyretin amyloidosis (ATTRv), a progressive, debilitating, and fatal disease that results from t…
View article: PS1221 LONG‐TERM EFFICACY AND SAFETY OF INOTERSEN FOR HEREDITARY TRANSTHYRETIN AMYLOIDOSIS: NEURO‐TTR OPEN‐LABEL EXTENSION 2‐YEAR UPDATE
PS1221 LONG‐TERM EFFICACY AND SAFETY OF INOTERSEN FOR HEREDITARY TRANSTHYRETIN AMYLOIDOSIS: NEURO‐TTR OPEN‐LABEL EXTENSION 2‐YEAR UPDATE Open
Background: Hereditary transthyretin amyloidosis (hATTR) is a rare protein misfolding disorder that causes progressive and debilitating polyneuropathy. A randomized, controlled phase 3 trial (NEURO‐TTR; NCT01737398) demonstrated efficacy a…
View article: P684Inotersen improved quality of life, polyneuropathy and cardiomyopathy in a diverse group of patients with hereditary transthyretin amyloidosis in the phase 3 study NEURO-TTR
P684Inotersen improved quality of life, polyneuropathy and cardiomyopathy in a diverse group of patients with hereditary transthyretin amyloidosis in the phase 3 study NEURO-TTR Open
View article: P685Hereditary transthyretin amyloidosis is associated with significant disease burden: analysis of the baseline characteristics of patients from the phase 3 study NEURO-TTR
P685Hereditary transthyretin amyloidosis is associated with significant disease burden: analysis of the baseline characteristics of patients from the phase 3 study NEURO-TTR Open
View article: Abstracts
Abstracts Open