Cambridge biotech Alnylam wins FDA approval for first drug

The FDA has approved Alnylam Pharmaceuticals Inc.'s first drug, a novel rare disease treatment that analysts believe could eventually top $1 billion in annual sales, marking a major milestone for one of the state's fastest-growing biotechs. The FDA said Friday it had approved Cambridge-based Alnylam's (Nasdaq: ALNY) drug patisiran, which targets a rare and often fatal genetic disease called hereditary ATTR amyloidosis, or hATTR amyloidosis. The disorder, which affects around 50,000 people worldwide,…
Source: Health Care:Pharmaceuticals headlines - Category: Pharmaceuticals Authors: Source Type: news

Related Links:

RARITAN, NJ, June 13, 2020 – The Janssen Pharmaceutical Companies of Johnson &Johnson announced today results from the first randomized Phase 3 study investigating subcutaneous daratumumab[i] in the treatment of patients with newly diagnosed light chain (AL) amyloidosis, a rare and potentially fatal disease.[1],[2] The data demonstrated subcutaneous daratumumab in combination with cyclophosphamide, bortezomib, and dexamethasone (D-CyBorD) resulted in a higher hematologic complete response rate (CR), (53 percent vs. 18 percent [P
Source: Johnson and Johnson - Category: Pharmaceuticals Tags: Innovation Source Type: news
This article is protected by copyright. All rights reserved.
Source: Journal of Cardiovascular Electrophysiology - Category: Cardiology Authors: Tags: EDITORIAL ‐ INVITED Source Type: research
Once considered a rare disease, wild type transthyretin amyloidosis (wtATTR) is currently being recognized as a prevalent disease in the elderly population, as it is estimated to be present in a significant subset of patients with heart failure with preserved ejection fraction (HFpEF) or with degenerative aortic stenosis [1,2]. The increasing population ageing, together with the availability of novel, non-invasive diagnostic tools (namely bone tracer scintigraphy), as well as the growing awareness in the medical community has led to a dramatic increase in the number of diagnosis of wtATTR in the developed countries [3].
Source: International Journal of Cardiology - Category: Cardiology Authors: Tags: Editorial Source Type: research
1366Objectives: CURRENT ROLE OF PET IMAGING IN CARDIAC AMYLOIDOSIS Cardiac amyloidosis (CA) is a relatively rare disease characterized by extracellular deposition of abnormal protein fibrils in the heart, resulting in restrictive cardiomyopathy and heart failure with preserved ejection fraction. Majority of CA occurs due to deposition of two types of amyloid protein: monoclonal immunoglobulin light chains (AL amyloidosis) or transthyretin (ATTR), either wild or mutant type. Differentiating the two most common types of amyloid deposition in the heart is of paramount importance in guiding therapy and prognostication. Diagnos...
Source: Journal of Nuclear Medicine - Category: Nuclear Medicine Authors: Tags: Cardiovascular Sciences (Poster Session) Source Type: research
This article will explore the etiology, those most commonly affected, types, appearance, treatment, prognosis, etc. Research began by focusing on amyloidosis in general then diving in further looking specifically at cardiac amyloid and PET amyloid imaging. Each article describes amyloidosis as a very rare disease that affects predominantly a male population between the ages of 30 and 80. There are several different types of amyloidosis that affect an array of organs including the heart, nerves, digestive system, and the kidneys. Nuclear Medicine, using small amounts of radiation, can help diagnose cardiac amyloidosis and a...
Source: Journal of Nuclear Medicine - Category: Nuclear Medicine Authors: Tags: Tech Students (Poster Session) Source Type: research
CONCLUSION: The diagnosis of amyloidosis is difficult because of its highly varied presentation. In case of clinical suspicion, a rapid, targeted diagnostic evaluation and subsequent initiation of treatment should be performed in a specialized center. When the new drugs to treat amyloidosis become commercially available, their use and effects should be documented in nationwide registries. PMID: 32295695 [PubMed - in process]
Source: Deutsches Arzteblatt International - Category: General Medicine Tags: Dtsch Arztebl Int Source Type: research
In this study, ...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
In Romania, 23 patients have been diagnosed with hereditary transthyretin amyloidosis (ATTRh), 18 of whom have the Glu54Gln mutation. This retrospective cohort included all patients with Glu54Gln-mutated ATTRh wh...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
CONCLUSION: PLOA can lead to visual and organ threatening complications. Accurate diagnosis is required for further diagnostic and therapeutic procedures and to counteract potential local and systemic complications. Interindividual differences in the course have to be considered. PMID: 31968364 [PubMed - in process]
Source: Klinische Monatsblatter fur Augenheilkunde - Category: Opthalmology Tags: Klin Monbl Augenheilkd Source Type: research
Hereditary gelsolin (AGel) amyloidosis is an autosomal dominantly inherited systemic amyloidosis that manifests with the characteristic triad of progressive ophthalmological, neurological and dermatological si...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
More News: Amyloidosis | Biotechnology | Genetics | Health Management | Pharmaceuticals | Rare Diseases