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The purpose of this study is to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of a single dose of ALN-TTR01 in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
Allocation: Randomized, Control: Placebo Control, Endpoint Classification: Safety Study, Intervention Model: Parallel Assignment, Masking: Single Blind (Subject), Primary Purpose: Treatment
Transthyretin Mediated Amyloidosis (ATTR)
ALN-TTR01, Sterile Normal Saline (0.9% NaCl)
Published on BioPortfolio: 2014-08-27T03:12:43-0400
The purpose of this study is to evaluate the safety and effectiveness of ALN-TTRSC in adults with transthyretin-mediated amyloidosis, whose disease has continued to worsen after liver tran...
The purpose of this study is to evaluate the safety and efficacy of long-term dosing with ALN-TTR02 (patisiran) in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
Hereditary (familial) amyloidosis arising from the misfolding of a mutated or variant transthyretin, is the most frequent form of amyloid cardiomyopathy in the Caribbean basin. Affected or...
This study is an online (web-based) or paper-based survey for patients with transthyretin familial amyloidosis polyneuropathy (TTR-FAP) and caregivers. The results will be used to describe...
The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).
Life-time diagnostics of wild type transthyretin amyloidosis (ATTR(wt)-amyloidosis) is virtually absent, even though ATTR(wt)-amyloidosis is an underestimated cause for morbidity and mortality, partic...
Hereditary transthyretin (ATTRm) amyloidosis is a rare, progressive and fatal disease with a range of clinical manifestations.
To describe abnormalities in choroidal and retinal vasculature associated with Val30Met familial transthyretin amyloidosis (V30M-FTA) using fluorescein and indocyanine green (ICG) angiography.
Gastrointestinal complications are common in hereditary transthyretin amyloid (ATTRm) amyloidosis. The underlying mechanisms have not been fully elucidated, and the patients' small bowel function rema...
The true prevalence of heart failure (HF) due to wild type transthyretin amyloidosis (ATTRwt) is likely underestimated. There is a paucity of data with regard to the management of ATTRwt-related advan...
Hypertonic sodium chloride solution. A solution having an osmotic pressure greater than that of physiologic salt solution (0.9 g NaCl in 100 ml purified water).
A form of CARDIAC MUSCLE disease in which the ventricular walls are excessively rigid, impeding ventricular filling. It is marked by reduced diastolic volume of either or both ventricles but normal or nearly normal systolic function. It may be idiopathic or associated with other diseases (ENDOMYOCARDIAL FIBROSIS or AMYLOIDOSIS) causing interstitial fibrosis.
A putative protein interaction module, approximately 70 amino acids long, that forms a small five-helix bundle with two large interfaces which may homo- and hetero-oligomerize, or bind non-sterile-alpha motif targets. The sterile alpha motif is present in a wide variety of eukaryotic proteins that function in diverse biological processes.
Diseases in which there is a familial pattern of AMYLOIDOSIS.
A genus of HALOBACTERIACEAE distinguished from other genera in the family by the presence of specific derivatives of TGD-2 polar lipids. Haloarcula are found in neutral saline environments such as salt lakes, marine salterns, and saline soils.