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Topic: PNH

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	Potential Effective Therapy For PNH, Improving Anaemia And Quality Of Life In Patients
		"PNH is a rare and life-threatening form of haemolytic anaemia where currently there are limited treatment options," said Dr. Peter Hillmen, senior author of the study, lead investigator and chairman of the TRIUMPH steering committee and Consultant Hematologist of the General Infirmary at the University of Leeds, Leeds, UK.
		PNH is characterized by severe destruction of red blood cells by the body's complement system (a component of the immune system).
		Importantly, PNH patients are at increased risk of forming life-threatening blood clots, or thromboses, which are the major cause of death.
	www.medicalnewstoday.com /medicalnews.php?newsid=52509&nfid=rssfeeds (0 words)

	eMedicine - Paroxysmal Nocturnal Hemoglobinuria : Article Excerpt by Emmanuel C Besa
		PNH currently is reclassified from purely an acquired hemolytic anemia due to a hematopoietic stem cell mutation defect.
		The classic description of the manifestations of PNH is the presence of dark urine during the night with partial clearing during the day (see Image 1); however, hemoglobinuria may occur every day in severe cases, more frequently in episodes lasting 3-10 days, or, in some cases, not at all.
		PNH may occur at any age, from children (10%) as young as 2 years to adults as old as 83 years, but it frequently is found among adults, with a median age at the time of diagnosis of 42 years (range, 16-75 y) from a series in England of 80 consecutive patients.
	www.emedicine.com /med/byname/paroxysmal-nocturnal-hemoglobinuria.htm (1171 words)

	News - Paper Reports Data on Eculizumab for the Treatment of PNH
		"PNH is a rare and life-threatening form of haemolytic anaemia where currently there are limited treatment options," said Dr. Peter Hillmen, senior author of the study, lead investigator and chairman of the TRIUMPH steering committee and Consultant Hematologist of the General Infirmary at the University of Leeds, Leeds, UK.
		PNH is characterized by severe destruction of red blood cells by the body's complement system (a component of the immune system).
		Importantly, PNH patients are at increased risk of forming life-threatening blood clots, or thromboses, which are the major cause of death.
	www.docguide.com /news/content.nsf/news/852571020057CCF6852571F00054BC63?OpenDocument&id=48DDE4A73E09A969852568880078C249&c=Anaemia&count=10 (654 words)

	Rare Thrombotic Diseases Consortium
		Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired disorder of hematopoiesis with an estimated incidence of 2 to 5 cases per million individuals in the United States.
		PNH may also occur in the setting of aplastic anemia or evolve into bone marrow aplasia (2;3).
		PNH is a clonal hematopoietic disorder, similar to myelodysplastic syndrome and chronic myelogenous leukemia.
	rarediseasesnetwork.epi.usf.edu /rtdc/physicians/pnh.htm (728 words)

	Paroxysmal nocturnal hemoglobinuria (Site not responding. Last check: )
		PNH is characterized by a decreased number of red blood cells (anemia), and the presence of blood in the urine (hemoglobinuria) and plasma (hemoglobinemia), which is evident after sleeping.
		PNH is associated with a high risk of major thrombotic events, most commonly thrombosis of large intra-abdominal veins.
		PNH blood cells are deficient in an enzyme known as PIG-A, which is required for the biosynthesis of cellular anchors.
	www.ncbi.nlm.nih.gov /books/bv.fcgi?call=bv.View..ShowSection&rid=gnd.section.97 (221 words)

	Eculizumab is well-tolerated and shows significant improvement in PNH patients (Ph III)
		PNH, a rare and life-threatening form of hemolytic anemia, is an acquired genetic blood disorder characterized by destruction of red blood cells by the body’s complement system (a component of the immune system).
		Importantly, PNH patients are at increased risk of forming life-threatening blood clots, or thromboses, which are a significant cause of death.
		Based upon scientific investigations and presentations of the prevalence of patients diagnosed with abnormal PNH cells in their blood, it is currently estimated that approximately 8,000 to 10,000 people in North America and Europe suffer from PNH.
	www.eurekalert.org /pub_releases/2006-12/erln-eiw120906.php (0 words)

	Biospace.com
		PNH is a blood disorder characterized by the onset of severe anemia, chronic fatigue and intermittent episodes of fl-colored urine, known as hemoglobinuria.
		PNH patients are also at increased risk of forming life-threatening blood clots, or thromboses, which are a leading cause of death (approximately 50%) in this disease.
		The mean percentage of PNH red blood cells increased from 36.7% of the total population found in the body to 59.2%, which helped reduce the mean patient transfusion rates from 2.1 units per patient, per month, to 0.6 units per patient, per month.
	www.biospace.com /news_story.cfm?StoryID=15062320&full=1 (938 words)

	Dr. Koop - Paroxysmal nocturnal hemoglobinuria (PNH)
		Paroxysmal nocturnal hemoglobinuria is a rare, acquired disorder of blood cells in whichÂ a specific molecule on the surface of the cells leads to premature destruction of the cells.
		PNH is caused by a defect in the formation of aÂ protein anchor called GPI.
		As a result of the lack of certain surface proteins, a number of surface proteins are unable to remain tethered to the cell surface, including the proteins CD55, also called Decay Accelerating Factor (DAF), and CD59, also called Membrane Inhibitor of Complement Lysis (MIRL).
	www.drkoop.com /ency/93/000534.html (384 words)

	HMDS: PNH Patient Information
		PNH occurs because one of the cells in the bone marrow (where blood is made) has undergone a change (or mutation).
		PNH is not an inherited condition (there is no increased risk in family members) and PNH cannot be passed from one person to another.
		Since PNH is an uncommon disease most patients with the disease may never have heard of it before they are diagnosed and would never meet another patient with PNH.
	www.hmds.org.uk /pnh_info.html (1120 words)

	Paper Reports Data on Eculizumab for the Treatment of PNH (Site not responding. Last check: )
		"PNH is a rare and life-threatening form of haemolytic anaemia where currently there are limited treatment options," said Dr. Peter Hillmen, senior author of the study, lead investigator and chairman of the TRIUMPH steering committee and Consultant Hematologist of the General Infirmary at the University of Leeds, Leeds, UK.
		PNH is characterized by severe destruction of red blood cells by the body's complement system (a component of the immune system).
		Importantly, PNH patients are at increased risk of forming life-threatening blood clots, or thromboses, which are the major cause of death.
	www.pslgroup.com /dg/264FFE.htm (608 words)

	Update: FDA Approves Alexion's Soliris(TM) For All Patients With PNH
		Patients with PNH are missing a specific protein that normally protects red blood cells from destruction by a component of the immune system called terminal complement.
		PNH is an acquired genetic blood disorder defined by hemolysis, in which patients' red blood cells are destroyed by complement, a component of the body's immune system.
		PNH is a rare disease that affects an estimated 8,000 to 10,000 people in North America and Europe.
	www.medilexicon.com /medicalnews.php?newsid=65557 (1363 words)

	AllRefer Health - Paroxysmal Nocturnal Hemoglobinuria (PNH) (PNH)
		Paroxysmal nocturnal hemoglobinuria is a rare, acquired disorder of red blood cells in which an abnormal cell surface molecule leads to premature destruction of the cells.
		PNH is caused by a defect in the formation of a red cell surface protein anchor, called GP1.
		As a result of the lack of this surface protein anchor, a number of surface proteins are unable to remain tethered to the cell surface.
	health.allrefer.com /health/paroxysmal-nocturnal-hemoglobinuria-pnh-info.html (0 words)

	Paroxysmal Nocturnal Hemoglobinuria (PNH): Anemias Caused by Hemolysis: Merck Manual Professional
		Paroxysmal nocturnal hemoglobinuria (PNH) is most common in men in their 20s, but it occurs in both sexes and at any age.
		PNH is suspected in patients who have typical symptoms of anemia or unexplained normocytic anemia with intravascular hemolysis, particularly if leukopenia or thrombocytopenia is present.
		If PNH is suspected, the sugar-water test is usually the first test performed; it relies on enhanced hemolysis of C3-dependent systems in isotonic solutions of low ionic strength, is simple to perform, and is sensitive.
	www.merck.com /mmpe/print/sec11/ch131/ch131c.html (435 words)

	Paroxysmal Nocturnal Hemoglobinuria (Site not responding. Last check: )
		Paroxysmal nocturnal haemoglobinuria (PNH) is characterized by the expansion of a haematopoietic stem cell clone with a PIG-A mutation (the PNH clone) in an environment in which normal stem cells are lost or failing: it has been hypothesized that this abnormal marrow environment provides a relative advantage to the PNH clone.
		PNH is caused by the expansion of a hematopoietic progenitor cell that caries a somatic mutation in the X-linked phosphatidylinositol glycan complementation group A (PIGA) gene.
		Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired hematopoietic stem cell disorder characterized by clonal blood cells that are deficient in glycosylphosphatidylinositol-anchored proteins because of somatic mutations of the PIG-A gene.
	www.thedoctorsdoctor.com /diseases/pnh.htm (8750 words)

	The Sidney Kimmel Comprehensive Cancer Center At Johns Hopkins
		PNH affects only 1-2 persons per million of the population and is a disease of young adults (median age of diagnosis 35-40 years of age) with occasional cases diagnosed in childhood or adolescence.
		Similarly, the risk developing PNH after treatment for aplastic anemia with immunosuppressive therapy (anti-thymocyte globulin and cyclosporine) is approximately 20 to 30%.
		PNH is caused when mutations of the PIG-A gene occur in a bone marrow stem cell.
	www.hopkinskimmelcancercenter.org /CANCERTYPES/paroxysmal-nocturnal-hemoglobinuria.cfm?CANCERID=341 (1035 words)

	CBBS: Transfusion of patients who have paroxysmal nocturnal hemoglobinuria (PNH)
		She indicates that her center currently supports two patients with PNH, and that her center is wondering if it would be safe to transfuse PNH patients with standard RBC units, rather than with saline-washed red cells.
		Interestingly, according to the Technical Manual editor, PNH patients frequently suffer GI bleeds (thought to be secondary to platelet abnormalities and a propensity for thrombosis), and are transfused with group-specific plasma without increases in hemolysis.
		In their last PNH patient who had episodic haemolysis, Buddi-Chiari syndrome, and a stem cell allograft, he did not have any haemolysis from leukodepleted RBCs (despite alleged haemolysis from unwashed RBC given in another hospital).
	www.cbbsweb.org /enf/2002/pnhtx_rbc.html (812 words)

	s050720a - Aplastic Anemia & Paroxysmal Nocturnal Hemoglobinuria (PNH)
		Abstract: To clarify the pathologic significance of granulocytes exhibiting the paroxysmal nocturnal haemoglobinuria (PNH) phenotype in patients with aplastic anaemia (AA), we examined peripheral blood from 100 patients with AA for the presence of granulocytes deficient in glycosylphosphatidylinositol (GPI)-anchored proteins using a sensitive flow cytometric assay.
		These findings suggest that a relative increase in the number of PNH granulocytes is a common feature of AA at diagnosis, and that it may represent the presence of immunologic pressure to normal haematopoietic stem cells as a cause of AA.
		Patients with PNH may not have an abnormal acid hemolysis test, and patients with aplastic anemia may present with evidence of abnormal sucrose lysis, acid hemolysis, and antibody-mediated complement hemolysis.
	www.emory.edu /WHSCL/grady/amreport/litsrch05/s050720a.html (2761 words)

	Genetic and environmental effects in paroxysmal nocturnal hemoglobinuria: this little PIG-A goes "Why? Why? Why?" -- ...
		Small deletion and insertion mutations (dark triangles) are most common in PNH and are distributed throughout the coding sequence; missense mutations (dark circles) appear to cluster in exon 2, suggesting that this region encodes for a functionally crucial portion of the protein (see ref. 29 for details of the GPI-anchor chemical structure).
		Deficiency of the GPl anchor caused by a somatic mutation of the PIG-A gene in paroxysmal nocturnal hemoglobinuria.
		Paroxysmal nocturnal haemoglobinuria (PNH) is caused by somatic mutations in the PIG-A gene.
	www.jci.org /cgi/content/full/106/5/637 (0 words)

	eculizumab New Drug Application
		An additional 11 PNH patients were enrolled in an earlier pilot study.
		Patients with PNH may suffer from severe hemolysis, anemia, chronic fatigue, recurrent pain, pulmonary hypertension and intermittent episodes of dark colored urine, known as hemoglobinuria.
		Importantly, PNH patients are at increased risk of forming life-threatening blood clots, or thromboses, which are a major cause of death in this disease.
	www.drugs.com /nda/soliris_061114.html (710 words)

	Disease > FDA Approves Alexion's Soliris for All Patients With PNH
		Soliris is indicated for the treatment of patients with PNH to reduce hemolysis.
		PNH often strikes people in the prime of their lives, with an average age of onset in the early 30's.
		The estimated median survival for PNH patients is between 10 and 15 years from the time of diagnosis.
	www.emaxhealth.com /39/10317.html (955 words)

	The PNH Source
		PNH causes a wide range of signs and symptoms—some of which are mild and others of which may be more severely disabling or life-threatening.
		Alexion is pleased to announce the approval of Soliris™ (eculizumab) for the treatment of all patients with PNH to reduce hemolysis.
		We are committed to expanding awareness and increasing the PNH patient and physician communities' knowledge about this debilitating and often life-threatening rare blood disease.
	www.pnhsource.com /index.php (0 words)

	PNH (Properties) Holdings PLC
		PNH (Properties) Residential Ltd dealing with the purchasing and rental of residential property.
		PNH (Properties) Commercial Ltd which owns and operates business centres in the South of England.
		PNH (Properties) Land Assembly Ltd which specialises in the process of assembling brownfield sites.
	www.pnhproperties.com (0 words)

	Alexion Pharmaceuticals, Inc. - Paroxysmal Nocturnal Hemoglobinuria (PNH)
		Paroxysmal nocturnal hemoglobinuria, or PNH, is a chronic disease where a a portion of a patient's oxygen-carrying red blood cells are missing the normally present complement inhibitors and are therefore abnormally fragile and inadvertently destroyed by normal complement activation.
		PNH is caused by a mutation in certain types of adult blood cells.
		Without these two protective proteins, PNH red blood cells, in particular, are easily burst by complement, resulting in low red blood cell count (anemia), fatigue, bouts of dark colored urine and various other complications.
	www.alexionpharm.com /f/20 (406 words)

	PNH Support Group
		If you have PNH or know someone who does, this group is a wonderful source of information.
		One of the great things about our members is that they are well informed about the disease and can either answer questions you have or put you in touch with experts who will be able to help.
		In 1999, she started a Yahoo Groups site so that she and the few others she had found with PNH could keep in touch, support each other, and share information.
	www.pnhdisease.org /index.php (301 words)

	Paroxysmal Nocturnal Hemoglobinuria (PNH) - Health Encyclopedia
		Paroxysmal nocturnal hemoglobinuria is a rare, acquired disorder of blood cells in which?a specific molecule on the surface of the cells leads to premature destruction of the cells.
		PNH is caused by a defect in the formation of a?protein anchor called GPI.
		As a result of the lack of certain surface proteins, a number of surface proteins are unable to remain tethered to the cell surface, including the proteins CD55, also called Decay Accelerating Factor (DAF), and CD59, also called Membrane Inhibitor of Complement Lysis (MIRL).
	www.nbcsandiego.com /encyclopedia/6860286/detail.html (611 words)

	An Interview with Patrick Nielsen Hayden
		PNH: It's hard to say what the beginnings of my career were because I have been involved in science fiction fandom, in fanzine publishing and convention organizing and so forth, for twenty-five years, since the mid-1970's at least.
		PNH: Actually my experience is that the bookstore buyers certainly can tell the difference and they will drop the numbers on the collection rather than on the next novel.
		PNH: The consulting structure, to some extent, is just an expression of Tom Doherty's desire to have a really great intake-scoop.
	www.sfwa.org /bulletin/articles/pnh_ds.html (0 words)

	News: Soliris(TM) (eculizumab) Demonstrates Significant Improvement in PNH Study. Genetic Engineering News - ...
		Patients with PNH lack naturally-occurring complement inhibitors on the surface of their blood cells which normally prevent red blood cell destruction.
		Patients with PNH may experience severe hemolysis (red blood cell destruction), anemia, chronic fatigue, recurrent pain, pulmonary hypertension and intermittent episodes of dark colored urine, known as hemoglobinuria.
		In January, 2006, Alexion announced that the first of those two PNH trials, the TRIUMPH study, achieved its co-primary endpoints with statistical significance.
	www.genengnews.com /news/bnitem.aspx?name=5808431&chid=4&taxid=29 (1470 words)

	Alexion Reports Results of Eculizumab Extension Trial in PNH in the Journal Blood - Health - RedOrbit
		PNH is a blood disorder characterized by the onset of severe anemia, chronic fatigue and intermittent episodes of dark colored urine, known as hemoglobinuria.
		PNH patients are also at increased risk of forming life- threatening blood clots, or thromboses, which are a leading cause of death (approximately 50%) in this disease.
		Lack of these complement inhibitor proteins leaves PNH red blood cells susceptible to destruction (hemolysis), which is manifest as a reduction in blood hemoglobin, causing patients to become anemic.
	www.redorbit.com /news/display?id=158808&source=r_health (1209 words)

	Clinical Trial: Eculizumab to Treat Paroxysmal Nocturnal Hemoglobinuria
		Paroxysmal nocturnal hemoglobinuria (PNH) is an acquired clonal disorder of the hematopoietic stem cell characterized by intravascular hemolysis, hemoglobinuria, anemia, and thrombosis.
		Evidence strongly suggests that lack of the terminal complement inhibitor CD59 is responsible for the sensitivity of PNH erythrocytes and platelets to the effects of autologous complement.
		Since the pathogenesis of PNH is due to the inability of PNH red cells and platelets to inhibit the activation of terminal complement, it is logical to hypothesize that a terminal complement inhibitor could effectively stop the intravascular hemolysis, obviate or lessen the need for transfusions, and possibly decrease the propensity of life threatening thrombosis.
	clinicaltrials.gov /ct/show/NCT00130000 (942 words)

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