The National Alliance of People with Rare Diseases is organizing a protest under the heading of “PROMISES DO NOT HEAL”. It will take place on September the 16th from 9 o’clock in front of the building of the National Assembly. For more information please contact Mr. Vladimir TOMOV (mobile phone +359 888 323 748).
An association of patients with primary immunodeficiencies is going to be created. The future organisation will defend the rights and the interests of the people with primary immunodeficiencies and their relatives. To be registrated officially and to work more efficiently is necessary to unite more people with this problem. If you are interested in, please contact Mr. Stoil LAZAROV (mobile phone: +359 899 999 698; e-mail: stoil_lazarov@abv.bg) or the Information Center for Rare Diseases and Orphan Drugs (phone/fax: +359-32/ 57 57 97; mobile phone: 0897 858 870; e-mail: info@raredis.org).
On 3/06/2008, a new orphan medicinal product was registered into the community register of orphan medicinal products under the EU orphan designation number 548. The active ingredient is Carfilzomib for treatment of multiple myeloma.
PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled Retroperitoneal fibrosis, M. Ormond, periaoartitis? ( Ther Umsch. 2008 May;65(5):265-8). Authors are Vogt B Meier P Burnier M. et al., from the Service de Néphrologie et d’Hypertension, Centre Médical Universitaire Vaudois (CHUV)-Université de Lausanne, Rue du Bugnon 17, Lausanne. Retroperitoneal fibrosis or Morbus Ormond is a rare disease characterized by inflammatory fibrosis of the retroperitoneal space and the abdominal aorta often including the common iliac arteries. The abdominal aorta can be enlarged leading to a classification of retroperitoneal fibrosis with or without aneurysm of the abdominal aorta. The classic form of retroperitoneal fibrosis usually presents without aneurysm of the abdominal aorta.Drug therapy is limited to steroids alone or in combination with immunosuppressive drugs such as azathioprine or even cyclophosphamide. The response to medical therapy is variable To access the full abstract of the article, click here.
On 3/06/2008, a new orphan medicinal product was registered into the community register of orphan medicinal products under the EU orphan designation number 549. The active ingredient is NGR-human tumour necrosis factor for treatment of malignant mesothelioma.
PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled Tracheopathia osteoplastica. (Ann Otolaryngol Chir Cervicofac. 2008 Jul 7). Authors are Liétin B Vellin JF Bivahagumye L et al., from the Service d’ORL et de chirurgie de la face et du cou, CHU de Clermont-Ferrand, BP 69, 63003 Clermont-Ferrand cedex 1, France. Report a case of tracheopathia osteoplastica and describe from a literature analysis the main clinical, radiological, and therapeutic features of this rare disease based on literature review.Tracheopathia osteoplastica is a rare tumor, with unknown etiology and physiopathology. The discovery is most often incidental. Progession is slow and it does not compromise the vital prognosis. The treatment is symptomatic. Few surgical tracheal operations are described in the literature. To access the full abstract of the article, click here.
PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled Progressive mitral valve thickening and progressive muscle cramps as manifestations of glycogenosis VII (Tarui’s Disease) (Cardiology. 2008;110(4):238-40). Authors are Finsterer J Stöllberger C from the Second Medical Department, and Krankenanstalt Rudolfstiftung, Vienna, Austria. Progressive heart valve thickening and shrinkage, and progressive muscle cramps have not been reported as manifestations of glycogenosis type VII (Tarui’s disease). In a 72-year-old female, Tarui’s disease was diagnosed in 1997, initially manifesting as simple partial seizures since 1977, anginal chest pain since 1982 and muscle cramps since 1983.Progression of Tarui’s disease may manifest as progressive thickening of the heart valves due to glycogen storage. Valve thickening may consecutively lead to valve insufficiency, enlargement of the atrium and atrial fibrillation. Progression of neurological manifestations may manifest as worsening muscle cramps. To access the full abstract of the article, click here.
On 3/06/2008, a new orphan medicinal product was registered into the community register of orphan medicinal products under the EU orphan designation number 550. The active ingredient is Nimotuzumab for treatment of pancreatic cancer.
PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled Ecallantide (DX-88), a plasma kallikrein inhibitor for the treatment of hereditary angioedema and the prevention of blood loss in on-pump cardiothoracic surgery (Expert Opin Biol Ther. 2008 Aug;8(8):1187-99). Author is Lehmann A from the Department of Anesthesiology and Intensive Care Medicine, Klinikum der Stadt Ludwigshafen, Germany. Plasma kallikrein plays a major role in the contact (kallikrein-kinin) cascade producing bradykinin. Bradykinin is a vasodilator, which increases vascular permeability, activates inflammation and produces pain. Plasma kallikrein is also crosslinked to the coagulation system and the complement cascade.Ecallantide (DX-88) is a potent and specific inhibitor of plasma kallikrein. Ecallantide is a recombinantly produced and engineered small protein. At present, the drug is being studied for two major indications. First, the results for the treatment of hereditary angioedema are promising. Second, a prospective randomised multi-centre trial for the reduction of blood loss during on-pump cardiothoracic surgery will be terminated in October 2008. To access the full abstract of the article, click here.
PubMed, the Internet portal of biomedical and life sciences literature, indexed an interesting article, entitled C1 inhibitor: just a serine protease inhibitor? New and old considerations on therapeutic applications of C1 inhibitor (Expert Opin Biol Ther. 2008 Aug;8(8):1225-40). Authors are Wouters D Wagenaar-Bos I van Ham M et al., from the Department of Immunopathology, Sanquin Research at CLB and Landsteiner Laboratory, University of Amsterdam, Academic Medical Center, Plesmanlaan 125, 1066 CX Amsterdam, The Netherlands. C1 inhibitor is a potent anti-inflammatory protein as it is the major inhibitor of proteases of the contact and the complement systems. C1-inhibitor administration is an effective therapy in the treatment of patients with hereditary angioedema (HAE) who are genetically deficient in C1 inhibitor. Owing to its ability to modulate the contact and complement systems and the convincing safety profile, plasma-derived C1 inhibitor is an attractive therapeutic protein to treat inflammatory diseases other than HAE. In the present review we give an overview of the biology of C1 inhibitor and its use in HAE. Furthermore, we discuss C1 inhibitor as an experimental therapy in diseases such as sepsis and myocardial infarction To access the full abstract of the article, click here.