Making Pharma Personal

Paul Perreault, CEO and Managing Director of biotherapies company CSL Behring, often receives messages from people celebrating the great landmarks in their lives. There is nothing so very extraordinary about that – except these are from patients whom he has helped during his time in the business. “I wasn’t always an executive, so I started in the business, and I know patients and their families personally, across the country,” he begins. “At different levels of jobs that I’ve had in the organization, I’ve interacted with patients on a very direct and one-on-one level. And I was around when there were shortages of products for patients, and patients were getting sick, and some died. And I was trying to access products for these patients - even from competitors, because it was about taking care of the patients. And today, I get pictures and postcards of them, their graduations from high school, and weddings, and these sorts of things.” Personal stake All this means that, while Perreault obviously drives CSL Behring’s patient-centric strategy at the executive level, there is something deeper going on for him. “I have a personal stake in this,” he says simply, in an interview ahead of his presentation at eyeforpharma Philadelphia. The company is a global leader in plasma protein biotherapeutics, which are used to treat serious and rare conditions such as hemophilia and von Willebrand disease, immune deficienci...
Source: EyeForPharma - Category: Pharmaceuticals Authors: Source Type: news

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DiscussionOur case series illustrates the difficulty in managing patients with anaphylaxis to replacement therapy. In the era of novel therapies, such as emicizumab, the management of HA patients who experience anaphylaxis to replacement therapy is becoming easier and may obviate the need for ITI. Current treatment strategies for HB patients with such anaphylaxis, however, are limited to rFVIIa, and it continues to pose a challenge.
Source: Blood Cells, Molecules, and Diseases - Category: Hematology Source Type: research
Condition:   Hemophilia A Intervention:   Procedure: Ultrasound, Haemophilia Joint Health Score Sponsors:   Swedish Orphan Biovitrum;   Kantar Health Not yet recruiting
Source: - Category: Research Source Type: clinical trials
CAMBRIDGE, Mass., Oct. 21, 2019 -- (Healthcare Sales &Marketing Network) -- SmartPharm Therapeutics, a preclinical-stage pharmaceutical company focused on developing next-generation, non-viral gene therapies for the treatment of rare diseases, today anno... Biopharmaceuticals, Personnel SmartPharm Therapeutics, gene therapy
Source: HSMN NewsFeed - Category: Pharmaceuticals Source Type: news
In this study, we present our contemporary experience in managing this rare condition from two major tertiary centers. This is a retrospective observational cohort study.
Source: Journal of Vascular Surgery - Category: Surgery Authors: Source Type: research
This report aimed to discuss the reason for IVCT in a case of pregnancy-induced hypertension with decreased antithrombin III and protein S and the anticoagulation strategy.
Source: Journal of Vascular Surgery - Category: Surgery Authors: Source Type: research
To investigate the von Willebrand factor to ADAMTS13 ratio as a potential biomarker for early detection of hepatocellular carcinoma (HCC) in cirrhosis.
Source: BMC Gastroenterology - Category: Gastroenterology Authors: Tags: Research article Source Type: research
ConclusionAccording to this study increasing the NO2 concentration has a direct and a significant effect on chronic obstructive pulmonary diseases. Electrical heating, alternative fuels solar and cooking are important factors in decreasing the concentration of NO2.
Source: Clinical Epidemiology and Global Health - Category: Epidemiology Source Type: research
Conclusion: Long-term OS and RFS after the surgical resection of primary chest wall sarcoma were clinically acceptable. PMID: 31624714 [PubMed]
Source: Korean Journal of Thoracic and Cardiovascular Surgery - Category: Cardiovascular & Thoracic Surgery Tags: Korean J Thorac Cardiovasc Surg Source Type: research
AbstractBoth transcatheter aortic valve implantation (TAVI) and surgical aortic valve replacement (SAVR) have been proven to effectively correct von Willebrand Factor (vWF) pathologies, however there is limited data simultaneously comparing outcomes of both approaches. We prospectively enrolled patients with severe aortic stenosis referred for TAVI (n  = 52) or SAVR (n = 48). In each case, vWF antigen (vWF:Ag), vWF activity (vWF:Ac) and activity-to-antigen (vWF:Ac/Ag) ratio were assessed at baseline, 24 h and 72 h after procedure. VWF abnormalities were defined as reduced vWF:Ac/Ag ratio (
Source: Journal of Thrombosis and Thrombolysis - Category: Hematology Source Type: research
Hemophilia B (HB, OMIM: 300746) is a rare X-linked recessive hemorrhagic disorder caused by the deficiency of coagulation Factor IX (FIX), with an estimated prevalence of 5.7 cases per 100,000 births [1]. The F9 gene, located on Xq27.1, comprises 8 exons and encodes the vitamin K-dependent FIX, a 461 amino acid precursor which is further cleaved and secreted into plasma as a mature, 415-residue activated protein. Upon activation, coagulation factor VIII (FVIII) and FIX form a tenase complex to activate coagulation factor X (FX) and the following coagulation pathway.
Source: Thrombosis Research - Category: Hematology Authors: Tags: Letter to the Editors-in-Chief Source Type: research
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