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Transthyretin (ATTR) amyloidosis: clinical spectrum, molecular pathogenesis and disease-modifying treatments.
J Neurol Neurosurg Psychiatry. 2015 Sep;86(9):1036-43. doi: 10.1136/jnnp-2014-308724. Epub 2015 Jan 20.
J Neurol Neurosurg Psychiatry. 2015.
PMID: 25604431
Review.
Analogous misfolding of wild-type TTR results in senile systemic amyloidosis, now termed wild-type ATTR amyloidosis, characterised by acquired amyloid disease in the elderly. ...Moreover, antisense oligonucleotides and small interfering RNAs for suppre …
Analogous misfolding of wild-type TTR results in senile systemic amyloidosis, now termed wild-type ATTR amyloidosis …
First nationwide survey on systemic wild-type ATTR amyloidosis in Japan.
Sekijima Y, Yazaki M, Ueda M, Koike H, Yamada M, Ando Y.
Sekijima Y, et al.
Amyloid. 2018 Mar;25(1):8-10. doi: 10.1080/13506129.2017.1409706. Epub 2017 Nov 28.
Amyloid. 2018.
PMID: 29182024
Clinical Trial.
OBJECTIVE: A nationwide survey on systemic wild-type ATTR (ATTRwt) amyloidosis was conducted to elucidate the frequency, clinical picture and possible diagnostic issues of ATTRwt amyloidosis in Japan. METHODS: A questionnaire was sent to 4629 clinical departments ac …
OBJECTIVE: A nationwide survey on systemic wild-type ATTR (ATTRwt) amyloidosis was conducted to elucidate the frequency, clinical pic …
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Tafamidis: a selective transthyretin stabilizer to treat wild-type ATTR amyloidosis and hereditary ATTR amyloidosis with cardiomyopathy.
Paton DM.
Paton DM.
Drugs Today (Barc). 2019 Dec;55(12):727-734. doi: 10.1358/dot.2019.55.12.3078389.
Drugs Today (Barc). 2019.
PMID: 31942875
This compound stabilizes TTR and has been found to significantly reduce the progression of both wild-type ATTR amyloidosis and hereditary ATTR amyloidosis....
This compound stabilizes TTR and has been found to significantly reduce the progression of both wild-type ATTR amylo …
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Safety and efficacy of a TTR specific antisense oligonucleotide in patients with transthyretin amyloid cardiomyopathy.
Benson MD, Dasgupta NR, Rissing SM, Smith J, Feigenbaum H.
Benson MD, et al.
Amyloid. 2017 Dec;24(4):219-225. doi: 10.1080/13506129.2017.1374946. Epub 2017 Sep 14.
Amyloid. 2017.
PMID: 28906150
Clinical Trial.
RESULTS: Eight patients with hereditary ATTR amyloidosis and 7 patients with wild-type ATTR amyloidosis with moderate to severely advanced restrictive cardiomyopathy showed stabilization of disease as measured by left ventricular wall thickness, left v …
RESULTS: Eight patients with hereditary ATTR amyloidosis and 7 patients with wild-type ATTR amyloidosis with mod …
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