Roche says Hemlibra effective in pivotal study

Roche's Hemlibra provided sustained bleed control in the largest pivotal study to date of children with a form of haemophilia, the Swiss drugmaker said on Monday.
Source: Reuters: Health - Category: Consumer Health News Tags: healthNews Source Type: news

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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
AbstractDamoctocog alfa pegol (Jivi®) is approved in the USA, EU, Japan and Canada for the treatment and prophylaxis of previously treated patients aged ≥ 12 years with haemophilia A. Formulated with a 60 kDa polyethylene glycol (PEG) moiety, damoctocog alfa pegol is an intravenously (IV) administered recombinant factor VIII (rFVIII) product with a longer terminal half-life than non-PEGylated FVIII and rFVIII products. In the multinational ph ase II/III PROTECT VIII trial, prophylaxis with damoctocog alfa pegol reduced the likelihood of bleeding in previously treated patients aged ≥ 1...
Source: Drugs - Category: Drugs & Pharmacology Source Type: research
This report presents a newborn case with severe hemophilia A, who had a splenic hematoma presented on the second day of life with severe anemia, abdominal distention, abdominal and scrotal ecchymosis. The patient was successfully treated medically with factor VIII concentrates without splenectomy. Molecular analysis of the factor VIII gene revealed a hemizygous deletion in exon 13.
Source: Transfusion and Apheresis Science - Category: Hematology Authors: Source Type: research
A clinical trial on hemophilia B demonstrated efficient liver targeting with AAV5 in the presence of neutralizing antibodies.
Source: Science Translational Medicine - Category: Biomedical Science Authors: Tags: Editors ' Choice Source Type: research
A new plasma donation center is set to open on July 10, at 222 Gadsden Highway in Birmingham.   OctaPharma is a Lachen, Switzerland-based company that allows people to donate plasma for medical production.  “OctaPharma converts donated plasma into plasma protein products used in more than 100 countries to treat life-threatening congenital and acquired diseases such as hemophilia A and B, immune deficiency syndromes, Rh disease in newborns and burn injuries,” Dr. David J. Aarons, medical director…
Source: Health Care:Biotechnology headlines - Category: Biotechnology Authors: Source Type: news
nnagl M Abstract Hemophilia A (HA) is an X-linked hereditary bleeding disorder caused by deficiency of coagulation factor (F) VIII activity. One of the greatest complications in the treatment of HA is the development of neutralizing alloantibodies, known as FVIII inhibitors. HA patients who develop FVIII inhibitors have limited treatment options available to them and experience greater disease- and treatment-related burdens than HA patients without FVIII inhibitors. Emicizumab, a recently approved bispecific monoclonal antibody, mimics the function of FVIIIa by bridging FIXa and FX to restore effective hemostasis....
Source: Thrombosis and Haemostasis - Category: Hematology Authors: Tags: Thromb Haemost Source Type: research
AbstractHemophilia is an inherited bleeding disorder which causes impaired blood clotting. The severity of disease depends on the type of Hemophilia, level of clotting factor concentrate (CFC), phenotypic heterogeneity and the development of inhibitors. The currently accepted standard of care of this disease is prophylaxis therapy (PT) with CFC. Prophylaxis therapy for Hemophilia is given in developed countries for the last few decades. On the contrary, episodic therapy (ET) is still the mode of treatment in middle and low income countries. ET is documented to have several potential risks such as increased bleeding rate, d...
Source: Indian Journal of Hematology and Blood Transfusion - Category: Hematology Source Type: research
Source: Patient Preference and Adherence - Category: International Medicine & Public Health Tags: Patient Preference and Adherence Source Type: research
Source: Expert Opinion on Biological Therapy - Category: Drugs & Pharmacology Authors: Source Type: research
Abstract Readthrough therapy relies on the use of small molecules that enable premature termination codons in mRNA open reading frames to be misinterpreted by the translation machinery, thus allowing the generation of full-length, potentially functional proteins from mRNAs carrying nonsense mutations. In patients with hemophilia A, nonsense mutations potentially sensitive to readthrough agents represent about 16% of the point mutations. The aim of this study was to measure the readthrough effect of different compounds and to analyze the influence of premature termination codon context in selected nonsense mutation...
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research
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