Pegcetacoplan ‐ a novel C3 inhibitor for paroxysmal nocturnal hemoglobinuria
<p dir="ltr">Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, life-threatening hematologic disorder due to clonal expansion of mutated bone marrow stem cells, in which their progeny lose the capacity to bind proteins to the cell surface. Chronic and/or paroxysmal intrava...
محفوظ في:
| المؤلف الرئيسي: | |
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| مؤلفون آخرون: | , , , |
| منشور في: |
2022
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| الموضوعات: | |
| الوسوم: |
إضافة وسم
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| _version_ | 1864513548320768000 |
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| author | Syeda Tayyaba Rehan (16897080) |
| author2 | Mahnoor Rehan Hashmi (17122952) Muhammad Sohaib Asghar (10395401) Muhammad Junaid Tahir (11654666) Zohaib Yousaf (9617058) |
| author2_role | author author author author |
| author_facet | Syeda Tayyaba Rehan (16897080) Mahnoor Rehan Hashmi (17122952) Muhammad Sohaib Asghar (10395401) Muhammad Junaid Tahir (11654666) Zohaib Yousaf (9617058) |
| author_role | author |
| dc.creator.none.fl_str_mv | Syeda Tayyaba Rehan (16897080) Mahnoor Rehan Hashmi (17122952) Muhammad Sohaib Asghar (10395401) Muhammad Junaid Tahir (11654666) Zohaib Yousaf (9617058) |
| dc.date.none.fl_str_mv | 2022-04-25T09:00:00Z |
| dc.identifier.none.fl_str_mv | 10.1002/hsr2.512 |
| dc.relation.none.fl_str_mv | https://figshare.com/articles/journal_contribution/Pegcetacoplan_a_novel_C3_inhibitor_for_paroxysmal_nocturnal_hemoglobinuria/29046065 |
| dc.rights.none.fl_str_mv | CC BY 4.0 info:eu-repo/semantics/openAccess |
| dc.subject.none.fl_str_mv | Biological sciences Genetics Biomedical and clinical sciences Cardiovascular medicine and haematology Pharmacology and pharmaceutical sciences Paroxysmal Nocturnal Hemoglobinuria (PNH) Hematologic Disorder Complement Inhibitors CD55 and CD59 Intravascular Hemolysis |
| dc.title.none.fl_str_mv | Pegcetacoplan ‐ a novel C3 inhibitor for paroxysmal nocturnal hemoglobinuria |
| dc.type.none.fl_str_mv | Text Journal contribution info:eu-repo/semantics/publishedVersion text contribution to journal |
| description | <p dir="ltr">Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, life-threatening hematologic disorder due to clonal expansion of mutated bone marrow stem cells, in which their progeny lose the capacity to bind proteins to the cell surface. Chronic and/or paroxysmal intravascular hemolysis and predilection to thrombosis are caused by the loss of the complement inhibitors CD55 and CD59 on the surface of red blood cells (RBC). A fraction of patients may also develop clinically severe aplastic anemia or myelodysplastic syndrome, as well as hypocellular or dysplastic bone marrow. PNH is characterized by fatigue, dysphagia, abdominal pain, dyspnea, dark urine, and erectile dysfunction. It predominantly affects Asians between 30 and 59 years. Therapy for PNH is evolving rapidly with the availability of biologic therapies that target the underlying complement-mediated hemolysis.</p><h2>Other Information</h2><p dir="ltr">Published in: Health Science Reports<br>License: <a href="http://creativecommons.org/licenses/by/4.0/" target="_blank">http://creativecommons.org/licenses/by/4.0/</a><br>See article on publisher's website: <a href="https://dx.doi.org/10.1002/hsr2.512" target="_blank">https://dx.doi.org/10.1002/hsr2.512</a></p> |
| eu_rights_str_mv | openAccess |
| id | Manara2_4a2f4f4371693068f27fce6d3f0ca46b |
| identifier_str_mv | 10.1002/hsr2.512 |
| network_acronym_str | Manara2 |
| network_name_str | Manara2 |
| oai_identifier_str | oai:figshare.com:article/29046065 |
| publishDate | 2022 |
| repository.mail.fl_str_mv | |
| repository.name.fl_str_mv | |
| repository_id_str | |
| rights_invalid_str_mv | CC BY 4.0 |
| spelling | Pegcetacoplan ‐ a novel C3 inhibitor for paroxysmal nocturnal hemoglobinuriaSyeda Tayyaba Rehan (16897080)Mahnoor Rehan Hashmi (17122952)Muhammad Sohaib Asghar (10395401)Muhammad Junaid Tahir (11654666)Zohaib Yousaf (9617058)Biological sciencesGeneticsBiomedical and clinical sciencesCardiovascular medicine and haematologyPharmacology and pharmaceutical sciencesParoxysmal Nocturnal Hemoglobinuria (PNH)Hematologic DisorderComplement InhibitorsCD55 and CD59Intravascular Hemolysis<p dir="ltr">Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, life-threatening hematologic disorder due to clonal expansion of mutated bone marrow stem cells, in which their progeny lose the capacity to bind proteins to the cell surface. Chronic and/or paroxysmal intravascular hemolysis and predilection to thrombosis are caused by the loss of the complement inhibitors CD55 and CD59 on the surface of red blood cells (RBC). A fraction of patients may also develop clinically severe aplastic anemia or myelodysplastic syndrome, as well as hypocellular or dysplastic bone marrow. PNH is characterized by fatigue, dysphagia, abdominal pain, dyspnea, dark urine, and erectile dysfunction. It predominantly affects Asians between 30 and 59 years. Therapy for PNH is evolving rapidly with the availability of biologic therapies that target the underlying complement-mediated hemolysis.</p><h2>Other Information</h2><p dir="ltr">Published in: Health Science Reports<br>License: <a href="http://creativecommons.org/licenses/by/4.0/" target="_blank">http://creativecommons.org/licenses/by/4.0/</a><br>See article on publisher's website: <a href="https://dx.doi.org/10.1002/hsr2.512" target="_blank">https://dx.doi.org/10.1002/hsr2.512</a></p>2022-04-25T09:00:00ZTextJournal contributioninfo:eu-repo/semantics/publishedVersiontextcontribution to journal10.1002/hsr2.512https://figshare.com/articles/journal_contribution/Pegcetacoplan_a_novel_C3_inhibitor_for_paroxysmal_nocturnal_hemoglobinuria/29046065CC BY 4.0info:eu-repo/semantics/openAccessoai:figshare.com:article/290460652022-04-25T09:00:00Z |
| spellingShingle | Pegcetacoplan ‐ a novel C3 inhibitor for paroxysmal nocturnal hemoglobinuria Syeda Tayyaba Rehan (16897080) Biological sciences Genetics Biomedical and clinical sciences Cardiovascular medicine and haematology Pharmacology and pharmaceutical sciences Paroxysmal Nocturnal Hemoglobinuria (PNH) Hematologic Disorder Complement Inhibitors CD55 and CD59 Intravascular Hemolysis |
| status_str | publishedVersion |
| title | Pegcetacoplan ‐ a novel C3 inhibitor for paroxysmal nocturnal hemoglobinuria |
| title_full | Pegcetacoplan ‐ a novel C3 inhibitor for paroxysmal nocturnal hemoglobinuria |
| title_fullStr | Pegcetacoplan ‐ a novel C3 inhibitor for paroxysmal nocturnal hemoglobinuria |
| title_full_unstemmed | Pegcetacoplan ‐ a novel C3 inhibitor for paroxysmal nocturnal hemoglobinuria |
| title_short | Pegcetacoplan ‐ a novel C3 inhibitor for paroxysmal nocturnal hemoglobinuria |
| title_sort | Pegcetacoplan ‐ a novel C3 inhibitor for paroxysmal nocturnal hemoglobinuria |
| topic | Biological sciences Genetics Biomedical and clinical sciences Cardiovascular medicine and haematology Pharmacology and pharmaceutical sciences Paroxysmal Nocturnal Hemoglobinuria (PNH) Hematologic Disorder Complement Inhibitors CD55 and CD59 Intravascular Hemolysis |