FDA OKs Inotersen for Hereditary ATTR With Polyneuropathy FDA OKs Inotersen for Hereditary ATTR With Polyneuropathy

Inotersen treatment led to improvements in neurologic and quality-of-life endpoints in patients with hereditary transthyretin amyloidosis with polyneuropathy in a pivotal phase 3 trial.FDA Approvals
Source: Medscape Medical News Headlines - Category: Consumer Health News Tags: Rheumatology News Alert Source Type: news

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Authors: AktaƟ A, Karadavut M, Cansu DÜ, Korkmaz C Abstract OBJECTIVES: To evaluate differences between the patients with familial Mediterranean fever (FMF) with homozygous (Hom), heterozygous (Het) and compound heterozygous (cHet) MEFV mutations in terms of clinical features and severity of the disease, as well as frequency of concomitant disorders, without focusing on Exon 10 mutations. METHODS: The patients with FMF were diagnosed using the Tel-Hashomer diagnostic criteria. The presence of MEFV mutations was investigated in exons 2,3,5 and 10 by multiplex-PCR reverse hybridisation method. All the patie...
Source: Clinical and Experimental Rheumatology - Category: Rheumatology Tags: Clin Exp Rheumatol Source Type: research
Authors: Ghersin I, Abu Alheija O, Azzam ZS, Nasser R PMID: 31599514 [PubMed - in process]
Source: The Israel Medical Association Journal - Category: General Medicine Tags: Isr Med Assoc J Source Type: research
We present the case of an 84-year-old man with chest tightness, dyspnoea, and ascites. He had a history of dyslipidaemia and ischaemic heart disease. Initial investigations showed severe diastolic dysfunction and elevated pulmonary artery systolic pressure on echocardiogram along with elevated serum natriuretic peptides. Further evaluation by a magnetic resonance imaging scan of the heart and endomyocardial biopsy confirmed the diagnosis of senile systemic amyloidosis. He made good progress after treatment with conventional heart failure drugs and is currently under consideration to start on specific medications to slow do...
Source: Journal of the Saudi Heart Association - Category: Cardiology Source Type: research
ConclusionsSurvival for patients with systemic AL amyloidosis has improved for patients at all stages of disease in the present era of rapid advancements in light chain-reducing therapies. Cardiac biomarkers at diagnosis, but not baseline dFLC ≥18 mg/dl, continue to provide important prognostic information.Central Illustration
Source: JACC: Heart Failure - Category: Cardiology Source Type: research
ConclusionOutpatient ASCT is a safe and feasible treatment strategy with low TRM. Overall resource utilization are significantly lower than inpatient auto-transplantation: however, this requires a multidisciplinary approach with close follow up
Source: Clinical Lymphoma Myeloma and Leukemia - Category: Cancer & Oncology Source Type: research
Abstract Cryopyrin-associated periodic syndrome (CAPS) is a group of rare hereditary autoinflammatory diseases characterised by recurrent flares of mild to severe systemic inflammation and fever. CAPS is the umbrella term for a spectrum of individual conditions, namely familial cold autoinflammatory syndrome (FCAS), Muckle-Wells syndrome (MWS) and neonatal-onset multisystem inflammatory disease (NOMID), also known as chronic infantile neurologic, cutaneous and articular (CINCA) syndrome. The flare symptoms include fever, fatigue, rashes, headaches, arthralgia and myalgia that can last for a few hours or for severa...
Source: British Journal of Nursing - Category: Nursing Authors: Tags: Br J Nurs Source Type: research
In this study, the effects of the plasma protein corona have been investigated with regard to the blood cell association and cytokine secretion of oligomeric (Aβo) and fibrillar Aβ1-42 (Aβf), two major forms of the peptide aggregates. Aβo displayed little change in membrane association in whole blood or washed blood (i.e., cells in the absence of plasma proteins) at 37 ºC, while Aβf showed a clear preference for binding with all cell types sans plasma proteins. Immune cells exposed to Aβo, but not to Aβf, resulted in significant expression of cytokines IL-6 and TNF measured in real-t...
Source: Biomacromolecules - Category: Biochemistry Authors: Tags: Biomacromolecules Source Type: research
Source: Journal of Nuclear Cardiology - Category: Nuclear Medicine Source Type: research
Conclusions In clinical practice, pimobendan seems to have beneficial effects in heart failure management for improving physical activities and the quality of life in patients with transthyretin cardiac amyloidosis. PMID: 31588087 [PubMed - as supplied by publisher]
Source: Internal Medicine - Category: Internal Medicine Tags: Intern Med Source Type: research
We present the case of an 84-years old man with chest tightness, dyspnoea and ascites. He had a history of dyslipidaemia and ischaemic heart disease. Initial investigations showed severe diastolic dysfunction and elevated PASP on echocardiogram along with elevated serum natriuretic peptides. Further evaluation by MRI scan of heart and endomyocardial biopsy confirmed the diagnosis of senile systemic amyloidosis. He made good progress after treatment with conventional heart failure drugs and is currently under consideration to start on specific medications to slow down the progression of amyloidosis. This case aims to increa...
Source: Journal of the Saudi Heart Association - Category: Cardiology Source Type: research
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