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37 results about "Von Willebrand disease" patented technology

An inherited bleeding disorder that results from low levels of specific clotting protein in blood.

Methods for correcting von willebrand factor point mutations

Some aspects of this disclosure provide strategies, systems, reagents, methods, and kits that are useful for the targeted editing of nucleic acids, including editing a nucleic acid encoding a mutant von Willebrand Factor protein to correct a point mutation associated with a disease or disorder, e.g., with von Willebrand disease. The methods provided are useful for correcting a vWF point mutation within the genome of a cell or subject, e.g., within the human genome. In some embodiments, fusion proteins of Cas9 and nucleic acid editing enzymes or enzyme domains, e.g., deaminase domains, are provided. In some embodiments, reagents and kits for the generation of targeted nucleic acid editing proteins, e.g., fusion proteins of Cas9 and nucleic acid editing enzymes or domains, are provided.
Owner:PRESIDENT & FELLOWS OF HARVARD COLLEGE

Liquid, Aqueous Pharmaceutical Composition of Factor VII Polypeptides

The present invention is directed to liquid, aqueous pharmaceutical compositions containing Factor VII polypeptides, and methods for preparing and using such compositions, as well as vials containing such compositions, and the use of such compositions in the treatment of a Factor VII-responsive syndrome, e.g., bleeding disorders, including those caused by clotting Factor deficiencies (e.g. haemophilia A, haemophilia B, coagulation Factor VII deficiency); by thrombocytopenia or von Willebrand's disease, or by clotting Factor inhibitors, and intra cerebral haemorrhage, or excessive bleeding from any cause. The preparations may also be administered to patients in association with surgery or other trauma or to patients receiving anticoagulant therapy. More particularly, the invention relates to liquid compositions stabilised against chemical and / or physical degradation. The main embodiment is represented by a liquid, aqueous pharmaceutical composition comprising a Factor VII polypeptide (i); a buffering agent (ii) suitable for keeping pH in the range of from about 4.0 to about 9.0; at least one metal-containing agent (iii), wherein said metal is selected from the group consisting of first transition series metals of oxidation state +II, except zinc, such as chromium, manganese, iron, cobalt, nickel, and copper; and a non-ionic surfactant (iv).
Owner:NOVO NORDISK HEALTH CARE AG

Preparation for treating hemophilia and von willebrand disease caused by heat stagnation blood aspect and method

The invention relates to a preparation for treating hemophilia and von willebrand disease caused by heat stagnation blood aspect and a method. The preparation is made of, by weight ratio, 6-15% of stir-fried cape jasmine fruit, 6-15% of tree peony bark, 6-15% of raw rhubarb, 18-48% of lalang grass rhizome, 6-15% of polygonum aviculare, 6-15% of Japanese thistle herb or root, 6-15% of common cephalanoplos herb, 9-24% of radix rubiae, 18-48% of hairyvein agrimonia herb and bud, 6-15 parts of Chinese arborvitae twig, 6-15% of carbonized catnip and 4-10% of radix glycyrrhizae. According to the preparation, a boiling extraction method is adopted to extract active ingredients in the raw medicines and keep activities thereof, so that not only can the dissolution rate of the active ingredients inthe medicines be improved, but also the absorption to the medicines can be facilitated, thereby improving the biological utilization degree thereof. In addition, the medicine is a pure traditional Chinese medicine compound preparation, so a patient does not produce toxic and side effects after long-term use, and the safety is good. In addition, the preparation method has a simple process and is convenient to operate.
Owner:天津市善济宏兴科技发展有限公司
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