Read by QxMD icon Read


Michael P Whyte, Katherine L Madson, Dawn Phillips, Amy L Reeves, William H McAlister, Amy Yakimoski, Karen E Mack, Kim Hamilton, Kori Kagan, Kenji P Fujita, David D Thompson, Scott Moseley, Tatjana Odrljin, Cheryl Rockman-Greenberg
Background. Hypophosphatasia (HPP) is caused by loss-of-function mutation(s) of the gene that encodes the tissue-nonspecific isoenzyme of alkaline phosphatase (TNSALP). Consequently, cell-surface deficiency of TNSALP phosphohydrolase activity leads to extracellular accumulation of inorganic pyrophosphate, a natural substrate of TNSALP and inhibitor of mineralization. Children with HPP can manifest rickets, skeletal pain, deformity, fracture, muscle weakness, and premature deciduous tooth loss. Asfotase alfa is a recombinant, bone-targeted, human TNSALP injected s...
June 16, 2016: JCI Insight
(no author information available yet)
This policy statement provides recommendations for the prevention of serogroup B meningococcal disease through the use of 2 newly licensed serogroup B meningococcal vaccines: MenB-FHbp (Trumenba; Wyeth Pharmaceuticals, a subsidiary of Pfizer, Philadelphia, PA) and MenB-4C (Bexsero; Novartis Vaccines, Siena, Italy). Both vaccines are approved for use in persons 10 through 25 years of age. MenB-FHbp is licensed as a 2- or 3-dose series, and MenB-4C is licensed as a 2-dose series for all groups. Either vaccine is recommended for routine use in persons 10 years and older who are at increased risk of serogroup B meningococcal disease (category A recommendation)...
September 2016: Pediatrics
Ernest U Ekpo, Suleman Bakhshi, Elaine Ryan, Peter Hogg, Mark F McEntee
The aim of this work was to examine the peak entrance surface air kerma (peak ESAK) to the eyes during CT fluoroscopy lung biopsy, and the impact of lead glasses, exposure parameters, head rotation, and height on peak ESAK to the eyes. Two phantoms simulating the patient and radiologist were used, and 108 exposures were made using a 16-slice Toshiba Alexion CT scanner (Toshiba Medical Systems, Nasu, Japan). ESAK to the phantom radiologist's right eye was measured using an Unfors Xi dosimeter (RaySafe, Billdal, Sweden) with and without lead glasses at two kilovoltages (120 kVp and 135 kVp) and three milliampere settings (10 mA, 20 mA, and 30 mA...
June 2016: Journal of Radiological Protection: Official Journal of the Society for Radiological Protection
Magnus M Berglund, Patrik Strömberg
No abstract text is available yet for this article.
2016: Expert Review of Proteomics
Matt Shirley
Sebelipase alfa (Kanuma™) is a recombinant human lysosomal acid lipase (LAL) developed by Synageva BioPharma Corp. (now Alexion Pharmaceuticals, Inc.) for long-term enzyme replacement therapy in patients with LAL deficiency. The agent, administered by intravenous infusion once weekly or once every other week, acts to replace the deficient enzyme activity in patients with LAL deficiency, reducing lysosomal lipid accumulation, and thereby improving disease-related abnormalities such as dyslipidaemia and liver abnormalities...
November 2015: Drugs
Mark Ratner
No abstract text is available yet for this article.
July 2015: Nature Biotechnology
Bernard S Kaplan, Rebecca L Ruebner, Joann M Spinale, Lawrence Copelovitch
Tremendous advances have been made in understanding the pathogenesis of atypical Hemolytic Uremic Syndrome (aHUS), an extremely rare disease. Insights into the molecular biology of aHUS resulted in rapid advances in treatment with eculizumab (Soliris(®), Alexion Pharmaceuticals Inc.). Historically, aHUS was associated with very high rates of mortality and morbidity. Prior therapies included plasma therapy and/or liver transplantation. Although often life saving, these were imperfect and had many complications...
May 2014: Intractable & Rare Diseases Research
Jun-ichi Nishimura, Masaki Yamamoto, Shin Hayashi, Kazuma Ohyashiki, Kiyoshi Ando, Andres L Brodsky, Hideyoshi Noji, Kunio Kitamura, Tetsuya Eto, Toru Takahashi, Masayoshi Masuko, Takuro Matsumoto, Yuji Wano, Tsutomu Shichishima, Hirohiko Shibayama, Masakazu Hase, Lan Li, Krista Johnson, Alberto Lazarowski, Paul Tamburini, Johji Inazawa, Taroh Kinoshita, Yuzuru Kanakura
BACKGROUND: Eculizumab is a humanized monoclonal antibody that targets complement protein C5 and inhibits terminal complement-mediated hemolysis associated with paroxysmal nocturnal hemoglobinuria (PNH). The molecular basis for the poor response to eculizumab in a small population of Japanese patients is unclear. METHODS: We assessed the sequences of the gene encoding C5 in patients with PNH who had either a good or poor response to eculizumab. We also evaluated the functional properties of C5 as it was encoded in these patients...
February 13, 2014: New England Journal of Medicine
C M Legendre, C Licht, P Muus, L A Greenbaum, S Babu, C Bedrosian, C Bingham, D J Cohen, Y Delmas, K Douglas, F Eitner, T Feldkamp, D Fouque, R R Furman, O Gaber, M Herthelius, M Hourmant, D Karpman, Y Lebranchu, C Mariat, J Menne, B Moulin, J Nürnberger, M Ogawa, G Remuzzi, T Richard, R Sberro-Soussan, B Severino, N S Sheerin, A Trivelli, L B Zimmerhackl, T Goodship, C Loirat
BACKGROUND: Atypical hemolytic-uremic syndrome is a genetic, life-threatening, chronic disease of complement-mediated thrombotic microangiopathy. Plasma exchange or infusion may transiently maintain normal levels of hematologic measures but does not treat the underlying systemic disease. METHODS: We conducted two prospective phase 2 trials in which patients with atypical hemolytic-uremic syndrome who were 12 years of age or older received eculizumab for 26 weeks and during long-term extension phases...
June 6, 2013: New England Journal of Medicine
Sean J Pittock, Vanda A Lennon, Andrew McKeon, Jay Mandrekar, Brian G Weinshenker, Claudia F Lucchinetti, Orna O'Toole, Dean M Wingerchuk
BACKGROUND: Complement activation after binding of an IgG autoantibody to aquaporin 4 (AQP4) is thought to be a major determinant of CNS inflammation and astrocytic injury in neuromyelitis optica. The aim of this study was to investigate the use of eculizumab--a therapeutic monoclonal IgG that neutralises the complement protein C5--in neuromyelitis optica spectrum disorders. METHODS: Between Oct 20, 2009, and Nov 3, 2010, we recruited patients from two US centres into an open-label trial...
June 2013: Lancet Neurology
Lucas Laursen
No abstract text is available yet for this article.
August 2011: Nature Biotechnology
S Chandran, L Baxter-Lowe, J L Olson, S J Tomlanovich, A Webber
A 34-year-old female recipient of a simultaneous pancreas-kidney transplant presented 7 days posttransplant with acute renal allograft dysfunction, thrombocytopenia, and microangiopathic hemolytic anemia. Renal biopsy revealed acute antibody-mediated rejection (AMR) and acute thrombotic microangiopathy (TMA). Clinical and laboratory manifestations, which had only partly responded to treatment with daily plasma exchange and intravenous immunoglobulin, resolved rapidly and completely to eculizumab (Soliris, Alexion Pharmaceuticals, Inc...
June 2011: Transplantation Proceedings
Amanda M Fitzpatrick, Cameron A Mann, Sarah Barry, Katie Brennan, James R Overell, Hugh J Willison
Human and animal studies on antibody-mediated neuropathy implicate complement in pathogenesis. In animal models complement inhibition is therapeutically beneficial. The monoclonal antibody, eculizumab (Soliris™, Alexion Pharmaceuticals, Cheshire, CT), prevents cleavage of C5 and thus inhibits terminal complement activation. In an open label study, 13 multifocal motor neuropathy patients received eculizumab for 14 weeks, 10 of whom were concomitantly receiving intravenous immunoglobulin. The primary outcome was safety of eculizumab, and the secondary outcomes included change in intravenous immunoglobulin (IVIg) dosing frequency, performance, and electrophysiological parameters...
June 2011: Journal of the Peripheral Nervous System: JPNS
Andrew Dmytrijuk, Kathy Robie-Suh, Martin H Cohen, Dwaine Rieves, Karen Weiss, Richard Pazdur
On March 16, 2007, eculizumab (Soliris; Alexion Pharmaceuticals, Inc. Cheshire, CT), a humanized monoclonal antibody that binds to the human C5 complement protein, received accelerated approval by the U.S. Food and Drug Administration for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) to reduce hemolysis. Eculizumab was studied in a randomized, double-blind, placebo-controlled clinical trial in 87 RBC transfusion-dependent adult PNH patients and in a supportive single-arm study in 96 patients...
September 2008: Oncologist
Jagruti A Patel, Somsuvra B Ghatak
Monoclonal antibodies are antibodies that are identical because they were produced by one type of immune cell, all clones of a single parent cell. This has become an important tool in biochemistry, molecular biology and medicine. The role of complement system in inflammation has been well established. Inflammation is a cornerstone of the post-myocardial infarction. Also, during a heart bypass procedure, the "complement activation" causes an inflammatory response that can lead to side effects such as chest pain, heart attack, stroke, heart failure, or death...
June 2008: Recent Patents on Cardiovascular Drug Discovery
Russell P Rother, Scott A Rollins, Christopher F Mojcik, Robert A Brodsky, Leonard Bell
The complement system provides critical immunoprotective and immunoregulatory functions but uncontrolled complement activation can lead to severe pathology. In the rare hemolytic disease paroxysmal nocturnal hemoglobinuria (PNH), somatic mutations result in a deficiency of glycosylphosphatidylinositol-linked surface proteins, including the terminal complement inhibitor CD59, on hematopoietic stem cells. In a dysfunctional bone marrow background, these mutated progenitor blood cells expand and populate the periphery...
November 2007: Nature Biotechnology
Lihua Bao, Richard J Quigg
The complement system consists of 3 pathways and more than 30 proteins, including those with biological activity that directly or indirectly mediate the effects of this system, plus a set of regulatory proteins necessary to prevent injudicious complement activation on host tissue. The role for complement in the pathogenesis of systemic lupus erythematosus (SLE) is paradoxic. On one hand, the complement system appears to have protective features in that hereditary homozygous deficiencies of classic pathway components are associated with an increased risk for SLE...
January 2007: Seminars in Nephrology
E G Spack
Within the past year a host of antigen-specific therapies for multiple sclerosis (MS) progressed along the path from IND submission to FDA approval. The Immune Response Corporation vaccinated patients with a Vbeta6 peptide, demonstrating that the vaccine was immunogenic, well tolerated, and reduced the number of Vbeta6+ T-cells in the cerebrospinal fluid (CSF). Connetics Corp. conducted a Phase I/II trial on chronic progressive MS patients vaccinated with CDR2 peptides from TCR Vbeta55.2 and found that patients with a measurable response to the vaccine remained clinically stable for a year...
November 1997: Expert Opinion on Investigational Drugs
Ani J Fleisig, Edward D Verrier
Myocardial injury and dysfunction in acute infarction and during cardiac surgery with cardiopulmonary bypass (CPB) are associated with an undesirable systemic inflammatory response, in which the complement cascade plays a major role. In animal models C5 inhibition has been found to significantly reduce myocardial infarct size and decrease cellular necrosis and apoptosis. Pexelizumab (Alexion Pharmaceuticals, Inc., Cheshire, CT, USA) is a humanized, monoclonal, single-chain antibody fragment that inhibits C5, thereby blocking its cleavage into active forms...
June 2005: Expert Opinion on Biological Therapy
Stanton K Shernan, Jane C K Fitch, Nancy A Nussmeier, John C Chen, Scott A Rollins, Christopher F Mojcik, Kevin J Malloy, Thomas G Todaro, Thomas Filloon, Steven W Boyce, Deepak M Gangahar, Michael Goldberg, Lawrence J Saidman, Dennis T Mangano et al.
BACKGROUND: During cardiac surgery requiring cardiopulmonary bypass, pro-inflammatory complement pathways are activated by exposure of blood to bio-incompatible surfaces of the extracorporeal circuit and reperfusion of ischemic organs. Complement activation promotes the generation of additional inflammatory mediators thereby exacerbating tissue injury. We examined the safety and efficacy of a C5 complement inhibitor for attenuating inflammation-mediated cardiovascular dysfunction in cardiac surgical patients undergoing cardiopulmonary bypass...
March 2004: Annals of Thoracic Surgery
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"