Roche to buy Spark Therapeutics

Roche Holding has entered a definitive merger agreement with Spark Therapeutics, as the Swiss drugmaker seeks to expand its ability to treat rare diseases through gene therapies, as well as build its hemophilia portfolio.
Source: PharmaManufacturing.com - Category: Pharmaceuticals Source Type: news

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This study reports the clinical characteristics and treatment outcomes of a relatively sizable cohort of patients with AHA. Methods: We retrospectively analyzed the characteristics and outcomes of 42 patients with AHA diagnosed in our center from January 2014 through December 2018. Results: The FVIII activity (FVIII: C) was significantly suppressed (median 1.5%; interquartile range [IQR]: 0.9-3.5) by FVIII inhibitor (median 8 BU/mL; IQR: 4.0-16.0). Bypassing agents, PCC or FVIIa, were used in 14 patients for bleeding control without any adverse reaction; and most patients (90.5%, 38/42) were placed on immunosuppressive reg...
Source: Hematology - Category: Hematology Tags: Hematology Source Type: research
Haemophilia A is a hereditary bleeding disorder, which has been considered rare and chronic. The burden of this disease in Portugal remains unknown. The aim of this study was to estimate the annualized cost an...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
ConclusionThough personalized treatment can be formalized to some extent, it is expected that medical treatment skills and management by tacit knowledge are affected in individualized treatment based on standardized treatment. Medical institutions and medical staff are the main body sharing and deploying measures to disseminate and utilize medical devices, cooperating with them can be an effective management of standardized and individualized treatment.
Source: Personalized Medicine Universe - Category: Drugs & Pharmacology Source Type: research
We present the first clinical description of a patient with superficial siderosis and hemophilia as the trigger of bleedings. Brain MRI was crucial for diagnosis.
Source: Interdisciplinary Neurosurgery - Category: Neurosurgery Source Type: research
CONCLUSION: Haemophilic patients went to the Emergency Department for common paediatric causes, but also requested consultation on specific problems related to haemophilia, with musculoskeletal problems/injury or bleeding being the main issues. The paediatric Emergency Department is an indispensable component of haemophilia care. PMID: 31171477 [PubMed - as supplied by publisher]
Source: Anales de Pediatria - Category: Pediatrics Authors: Tags: An Pediatr (Barc) Source Type: research
An external jugular vein (EJV) aneurysm is an extremely rare disease. Ultrasonography with color doppler methods is considered the criterion standard for diagnosis of a venous aneurysm. The treatment of choice for an EJV aneurysm is surgical excision. Herein, we report an unusual case of an EJV aneurysm in a child with hemophilia A. Because the incidence of an EJV aneurysm is low, it is important to distinguish it from other neck lesions, including congenital disease. In addition, it should be noted that in hemophilia patients, an EJV aneurysm may occur in a spontaneous or small trauma.
Source: Journal of Craniofacial Surgery - Category: Surgery Tags: Brief Clinical Studies Source Type: research
Reena Goswami1, Gayatri Subramanian2, Liliya Silayeva1, Isabelle Newkirk1, Deborah Doctor1, Karan Chawla2, Saurabh Chattopadhyay2, Dhyan Chandra3, Nageswararao Chilukuri1 and Venkaiah Betapudi1,4* 1Neuroscience Branch, Research Division, United States Army Medical Research Institute of Chemical Defense, Aberdeen, MD, United States 2Department of Medical Microbiology and Immunology, University of Toledo College of Medicine and Life Sciences, Toledo, OH, United States 3Roswell Park Comprehensive Cancer Center, Buffalo, NY, United States 4Department of Physiology and Biophysics, Case Western Reserve University, Clev...
Source: Frontiers in Oncology - Category: Cancer & Oncology Source Type: research
This study sought to identify hemophilia carriers in C ôte d’Iv...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
In this study, the potential of PF-06741086 to restore thrombin generation in rare disease plasma was explored.Thrombin generation was measured in citrated platelet poor Factor XI (FXI), Factor V (FV), FVII, von Willebrand Factor (vWF) deficient (Type 1, 2A, 2B and 3) congenital donor plasma following the in vitro addition of PF-06741086 (0, 1, 10 or 100 nM) or a human IgG1 antibody; initiated with 1 pM TF and 4 µM phospholipid. FXI, FV, and FVII donors had less than 1% coagulation factor activity. Non-hemophilic plasma from healthy donors alone was also included in the analysis. In FXI deficient plasmas, a concentra...
Source: Blood - Category: Hematology Authors: Tags: 321. Blood Coagulation and Fibrinolytic Factors: Poster II Source Type: research
Rare disease drug company BioMarin put on a show called ‘Hemophilia: The Musical’ performed by teenagers affected by blood disorders.
Source: Forbes.com Healthcare News - Category: Pharmaceuticals Authors: Source Type: news
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