Vyndaqel Capsules Special Investigation (ATTR-CM)

2019-10-03 07:54:43 | BioPortfolio


Secondary Data Collection : To confirm the safety and effectiveness profiles under the actual medical practice of Vyndaqel in Japan.


To comprehend information on the long-term safety (e.g., onset status of adverse reactions), etc. of patients who are treated with Vyndaqel for the treatment of transthyretin amyloid cardiomyopathy.

Study Design


Transthyretin (TTR) Amyloid Cardiomyopathy


Treatment for TTR amyloidosis




Not yet recruiting



Results (where available)

View Results


Published on BioPortfolio: 2019-10-03T07:54:43-0400

Clinical Trials [545 Associated Clinical Trials listed on BioPortfolio]

Mitochondrial Function in Transthyretin Amyloidosis

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...

Long-term Safety of Tafamidis in Subjects With Transthyretin Cardiomyopathy

Phase 3 extension study to evaluate the safety of oral daily dosing of 20 mg or 80 mg tafamidis meglumine in subjects with either transthyretin genetic variants or wild-type transthyretin ...

HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

This study will evaluate the efficacy and safety of vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) compared to placebo in patients with ATTR amyloidosis with c...

Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy

Phase 3 efficacy and safety study to evaluate AG10 800 mg compared to placebo in subjects with symptomatic Transthyretin Amyloid Cardiomyopathy (ATTR-CM)

APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

The purpose of this study is to evaluate the efficacy and safety of patisiran in participants with ATTR amyloidosis with cardiomyopathy.

PubMed Articles [17459 Associated PubMed Articles listed on BioPortfolio]

Amyloid cardiomyopathy in a large integrated health care system.

Light Chain (AL) and transthyretin (ATTR) amyloidosis are the most common forms of amyloid cardiomyopathy. Population based studies describing the epidemiology and clinical features of amyloid cardiom...

Transthyretin cardiac amyloidosis: an update on diagnosis and treatment.

Transthyretin cardiac amyloidosis (ATTR-CA) demonstrates progressive, potentially fatal, and infiltrative cardiomyopathy caused by extracellular deposition of transthyretin-derived insoluble amyloid f...

Neuropathy Associated with Systemic Amyloidosis.

Peripheral neuropathy occurs in the setting of both hereditary and acquired amyloidosis. The most common form of hereditary amyloidosis is caused by 1 of 140 mutations in the transthyretin (TTR) gene,...

Transthyretin Amyloidosis: Putting Myopathy On The Map.

While peripheral neuropathy and cardiomyopathy are well recognized manifestations of transthyretin (ATTR) amyloidosis, myopathy has been rarely reported.

Estimating the Prevalence of Transthyretin Amyloid Cardiomyopathy in a Large In-Hospital Database in Japan.

Transthyretin amyloid cardiomyopathy (ATTR-CM)-a debilitating, fatal disease resulting from the deposition of transthyretin (TTR) amyloid fibrils-can be hereditary due to mutations in the TTR gene (AT...

Medical and Biotech [MESH] Definitions

A nonproliferative disorder of the PLASMA CELL characterized by excessive production and misfolding of IMMUNOGLOBULIN LIGHT CHAINS that form insoluble amyloid fibrils (see AMYLOID DEPOSITS) in various tissues. Clinical features include LIVER FAILURE; MULTIPLE MYELOMA; NEPHROTIC SYNDROME; RESTRICTIVE CARDIOMYOPATHY, and neuropathies.

Inherited disorders of the peripheral nervous system associated with the deposition of AMYLOID in nerve tissue. The different clinical types based on symptoms correspond to the presence of a variety of mutations in several different proteins including transthyretin (PREALBUMIN); APOLIPOPROTEIN A-I; and GELSOLIN.

An ACUTE PHASE REACTION protein present in low concentrations in normal sera, but found at higher concentrations in sera of older persons and in patients with AMYLOIDOSIS. It is the circulating precusor of amyloid A protein, which is found deposited in AA type AMYLOID FIBRILS.

A group of sporadic, familial and/or inherited, degenerative, and infectious disease processes, linked by the common theme of abnormal protein folding and deposition of AMYLOID. As the amyloid deposits enlarge they displace normal tissue structures, causing disruption of function. Various signs and symptoms depend on the location and size of the deposits.

A heterogeneous group of sporadic or familial disorders characterized by AMYLOID deposits in the walls of small and medium sized blood vessels of CEREBRAL CORTEX and MENINGES. Clinical features include multiple, small lobar CEREBRAL HEMORRHAGE; cerebral ischemia (BRAIN ISCHEMIA); and CEREBRAL INFARCTION. Cerebral amyloid angiopathy is unrelated to generalized AMYLOIDOSIS. Amyloidogenic peptides in this condition are nearly always the same ones found in ALZHEIMER DISEASE. (from Kumar: Robbins and Cotran: Pathologic Basis of Disease, 7th ed., 2005)

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