Cambridge's Akcea rebounds with FDA approval of first drug

After having one of its drugs rejected by the FDA in August, Cambridge biotech Akcea Therapeutics Inc. turned the tide on Friday, receiving approval for another treatment for a rare genetic disease. The FDA approved Tegsedi, known generically as inotersen, an RNA-based drug that is designed to treat a rare and often fatal genetic disease called hereditary ATTR amyloidosis, or hATTR amyloidosis. According to Akcea (Nasdaq: AKCA), the drug works by slowing down production of a protein that can build…
Source: bizjournals.com Health Care:Physician Practices headlines - Category: American Health Authors: Source Type: news

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Publication date: Available online 8 April 2020Source: Respiratory Medicine Case ReportsAuthor(s): Thiyaphat Laohawetwanit, Kei Tanaka, Yoshiaki Zaizen, Kazuhiro Tabata, Kouji Ando, Hiroshi Ishimoto, Hiroshi Mukae, Yasushi Miyazaki, Andrey Bychkov, Junya Fukuoka
Source: Respiratory Medicine Case Reports - Category: Respiratory Medicine Source Type: research
No abstract available
Source: European Journal of Gastroenterology and Hepatology - Category: Gastroenterology Tags: Letters to the Editor Source Type: research
Light chain deposition disease (LCDD) is commonly associated with monoclonal gammopathy of renal significance (MGRS). Venetoclax is a Bcl-2 inhibitor approved for the treatment of chronic lymphocytic leukemia with activity against multiple myeloma and AL amyloidosis clones that harbor the t(11;14) abnormality. Here, we report the first use of venetoclax in a patient with LCDD secondary to MGRS after kidney transplantation. A 43 year-old male presented with 17.9 g/d of proteinuria and creatinine of 3.7 mg/dl.
Source: Clinical Lymphoma, Myeloma and Leukemia - Category: Hematology Authors: Tags: Case Report Source Type: research
No concerted investigation has been conducted to explore overlapping and distinct pathobiological mechanisms between repetitive mild traumatic brain injury (r-mTBI) and tau/amyloid proteinopathies considering the long history of association between TBI...
Source: SafetyLit - Category: International Medicine & Public Health Tags: Ergonomics, Human Factors, Anthropometrics, Physiology Source Type: news
AbstractCerebral amyloid angiopathy-related inflammation is a syndrome of reversible encephalopathy with cerebral amyloid angiopathy, however the pathology is not well understood. We clear a part of the pathology through the first case of an 80-year-old man with cerebral amyloid angiopathy-related inflammation induced by relapsing polychondritis (RP) analysis. An 80-year-old man was diagnosed with RP by auricular cartilage biopsy. Almost no abnormality including intracranial microbleeding was detected by cranial magnetic resonance image (MRI) at diagnosis. However, he developed a headache and hallucination after five month...
Source: Journal of Thrombosis and Thrombolysis - Category: Hematology Source Type: research
SO Abstract Autoinflammatory syndromes (AIS) are characterized by uniform attacks often with febrile episodes, exanthema, abdominal pain, muscle and joint pain. Patients show markedly elevated levels of the inflammatory serum parameters C‑reactive protein (CRP) and systemic amyloid A (SAA) during an attack. The origin of the family of the patient and the duration of the attacks are helpful to find the appropriate diagnosis. Molecular genetic tests are used to confirm the clinical diagnosis of an AIS. Colchicine can prevent attacks of familial Mediterranean fever but not the other forms of AIS. In refractory cas...
Source: Zeitschrift fur Rheumatologie - Category: Rheumatology Authors: Tags: Z Rheumatol Source Type: research
We present the case of a young female patient diagnosed through kidney biopsy with amyloid A (AA) amyloidosis after 30 years of evolution of FMF and we review the present knowledge regarding the pathogenesis and management of this rare genetic disease. PMID: 32239108 [PubMed - in process]
Source: Romanian Journal of Morphology and Embryology - Category: General Medicine Tags: Rom J Morphol Embryol Source Type: research
Publication date: April 2020Source: Journal of Comparative Pathology, Volume 176Author(s): C. Bianco, P.J. Sánchez-Cordón, R. Verin, A. Godinho, U. Weyer, S. Lesellier, J. Spiropoulos, T. Floyd, D. Everest, A. Núñez
Source: Journal of Comparative Pathology - Category: Pathology Source Type: research
Elucidation of the biologic mechanisms underlying the pathogenesis of transthyretin (TTR) amyloidosis has led to the approval of therapies to treat both inherited (hATTR) and age-related or wild-type (wtATTR) forms of transthyretin amyloidosis manifesting as neuropathy and/or cardiomyopathy1-3. These developments have been hailed as a model of scientific and medical progress emanating from precision medicine, which can arrest or slow progression of fatal disorders and provide a potential pathway for progress in other complex clinical syndromes such as heart failure with preserved ejection fraction by endo-phenotyping.
Source: The American Journal of Medicine - Category: General Medicine Authors: Tags: Commentary Source Type: research
(Shinshu University) Small gauge vitrectomy for vitreous amyloidosis and subsequent management of secondary glaucoma in patients with hereditary transthyretin amyloidosis.
Source: EurekAlert! - Medicine and Health - Category: International Medicine & Public Health Source Type: news
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