What you missed on your summer vacation in Mass. biotech news

Here's a look at the Boston biotech news you missed during your summer vacation: Alnylam gets historic FDA approval for first drug Cambridge's Alnylam Pharmaceuticals (Nasdaq: ALNY) received FDA approval for its first drug on Aug. 10, a first-ever treatment for a rare genetic disease that uses RNA interference to silence genes. The drug treats hereditary ATTR amyloidosis, w hich causes the liver to protein abnormal proteins that impede nervous system and organ functions. Marketed under the name…
Source: bizjournals.com Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news

Related Links:

Condition:   Transthyretin Amyloidosis (ATTR) With Cardiomyopathy Interventions:   Drug: Placebo;   Drug: Patisiran Sponsor:   Alnylam Pharmaceuticals Not yet recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Constrictive Pericarditis (CP) is a curable and reversible form of severe diastolic heart failure. We aimed to investigate the diagnostic accuracy of published echocardiographic Mayo Clinic Criteria in differentiating in 107 patients with surgically proven CP from 30 patients with Restrictive Cardiomyopathy (RCM) due to cardiac Amyloidosis. Five principal echocardiographic and Doppler variables were re-measured on preoperative transthoracic echocardiogram namely: 1) respiration-related ventricular septal shift; 2) respiratory variation in mitral inflow E pulsed Doppler velocity 3) tissue Doppler medial mitral annular e &rs...
Source: The American Journal of Cardiology - Category: Cardiology Authors: Source Type: research
Immunoglobulin light chain amyloidosis is the most frequent type of renal amyloidosis in the United States, accounting for 81% of cases. Accurate typing is crucial for early diagnosis and treatment of immunoglobulin-derived amyloidosis and to avoid treating other amyloidoses with potentially toxic chemotherapy. Immunofluorescence is the first step to type renal immunoglobulin-derived amyloidosis but the performance characteristics of this method are largely unknown. Here, we establish the sensitivity and specificity of immunofluorescence for diagnosing immunoglobulin-derived amyloidosis in patients whose amyloid typing was...
Source: Kidney International - Category: Urology & Nephrology Authors: Tags: Clinical Investigation Source Type: research
Conditions:   Amyloidosis Cardiac;   Carpal Tunnel Syndrome Intervention:   Diagnostic Test: DPD Scintigraphy Sponsors:   Aarhus University Hospital Skejby;   Regional Hospital Holstebro Recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
Publication date: Available online 22 June 2019Source: Journal of Photochemistry and Photobiology B: BiologyAuthor(s): Nora Alkudaisi, Ben Allan Russell, David J.S. Birch, Yu ChenAbstractProtein aggregation can lead to several incurable amyloidosis diseases. The full aggregation pathway is not fully understood, creating the need for new methods of studying this important biological phenomenon. Lysozyme is an amyloidogenic protein which is often used as a model protein for studying amyloidosis. This work explores the potential of employing Lysozyme encapsulated gold nanoclusters (Ly-AuNCs) to study the protein's aggregation...
Source: Journal of Photochemistry and Photobiology B: Biology - Category: Speech-Language Pathology Source Type: research
Conditions:   Amyloidosis Cardiac;   Carpal Tunnel Syndrome Intervention:   Diagnostic Test: DPD Scintigraphy Sponsors:   Aarhus University Hospital Skejby;   Regional Hospital Holstebro Recruiting
Source: ClinicalTrials.gov - Category: Research Source Type: clinical trials
We describe the limitations of cardiac biopsies without mass spectrometry in subtyping.
Source: Heart, Lung and Circulation - Category: Cardiology Authors: Tags: 068 Source Type: research
CONCLUSION: In patients with MCN, %UAE may be a useful marker for the detection of coexistence of glomerular lesions, such as amyloidosis, which are associated with a poor outcome.
. PMID: 31232268 [PubMed - as supplied by publisher]
Source: Clinical Nephrology - Category: Urology & Nephrology Authors: Tags: Clin Nephrol Source Type: research
Background: Cardiac amyloidosis manifests as a progressive infiltrative cardiomyopathy for which delayed diagnosis is common and predicts a poor prognosis. Additionally, right ventricular dysfunction has been a predictor of mortality in cardiac amyloidosis. Strain imaging has been shown to detect subclinical cardiac dysfunction in a range of cardiomyopathies.
Source: Heart, Lung and Circulation - Category: Cardiology Authors: Tags: 317 Source Type: research
3,3-diphosphono-1,2-propanodicarboxylic acid (DPD) is the most validated tracer for detecting cardiac amyloidosis with bone scintigraphy. PYP and HDP are more readily available in Australia, and considered possible alternatives. We sought to examine experience using HDP and PYP at the Victorian and Tasmanian Amyloidosis Service.
Source: Heart, Lung and Circulation - Category: Cardiology Authors: Tags: 308 Source Type: research
More News: Amyloidosis | Biotechnology | Genetics | Health Management | Liver | Pharmaceuticals | Urology & Nephrology