Alnylam Announces FDA Acceptance of New Drug Application (NDA) and Priority Review Status for Patisiran, an Investigational RNAi Therapeutic for the Treatment of Hereditary ATTR (hATTR) Amyloidosis

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Feb. 1, 2018-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing its New Drug Application...
Source: Drugs.com - New Drug Applications - Category: Drugs & Pharmacology Source Type: news

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Publication date: Available online 14 October 2019Source: Respiratory Medicine Case ReportsAuthor(s): Chunli Che, Fushi Dong, Xue Wu, Wei Wang, Lixue JiangAbstractObjectiveThis case report shows that bronchoscopy is an important method to treat severe airway stenosis caused by bronchial amyloidosis. Bronchoscopic forceps were used to incise the intra-tracheal lump repeatedly. The incision was frozen with a cryosurgery probe, argon knife was used to stop the bleeding until the airway lumen stenosis was reduced to approximately 40%, after which, it continued to enter the lumen. We used bronchoscopic biopsy forceps to repeate...
Source: Respiratory Medicine Case Reports - Category: Respiratory Medicine Source Type: research
Researchers at the University of Turku have discovered new changes in blood samples associated with Alzheimer’s disease. A new study was conducted on disease-discordant Finnish twin pairs: one sibling suffering from Alzheimer's disease and the other being cognitively healthy. The researchers used the latest genome-wide methods to examine the twins' blood samples for any disease-related differences in epigenetic marks which are sensitive to changes in environmental factors. These differences between the siblings were discovered in multiple different genomic regions. Development of the late-onset form of Al...
Source: MDDI - Category: Medical Devices Authors: Tags: R & D Source Type: news
Continuous positive airway pressure (CPAP) is a first-line treatment for obstructive sleep apnea (OSA). However, the adherence to CPAP can be difficult for patients due to several reasons. In this case report we describe a patient with severe OSA who initially was treated successfully with CPAP, but after a few months adherence problems developed because of macroglossia. This was caused by amyloidosis due to multiple myeloma. During treatment with chemotherapy and stem cell transplantation, the size of the patient’s tongue decreased and he could successfully use his CPAP device again.Citation:van Loenhout L, van der ...
Source: Journal of Clinical Sleep Medicine : JCSM - Category: Sleep Medicine Source Type: research
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
The diagnosis of primary systemic amyloidosis, also known as AL (amyloid light-chain) amyloidosis, is often delayed owing to its nonspecific manifestations as well as its rarity. A 64-year-old woman presented with an eight-month history of significant weight loss, anemia, fatigue, and progressive painful cutaneous lesions on her hands, lips, back, perianal, and vulvar area that were originally treated unsuccessfully with antimalarials and systemic corticosteroids. Histopathological examination revealed an amorphous dermis with pale pink material that demonstrated positive birefringence with Congo red staining. Subsequently...
Source: Dermatology Online Journal - Category: Dermatology 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
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