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1. Full Text Rapamycin and rapalogs for tuberous sclerosis complex
by Sasongko, Teguh H and Ismail, Nur Farrah Dila and Zabidi‐Hussin, ZAMH and Sasongko, Teguh H
Cochrane Database of Systematic Reviews, ISSN 1469-493X, 07/2016, Volume 2017, Issue 6, pp. CD011272 - CD011272
OTHER TOPICS | Kidney Neoplasms | Other blood disorders | Immunosuppressive Agents | Genetic disorders | Sirolimus | Administration, Oral | Tuberous Sclerosis | Skin Diseases | Haemophilia & other coagulopathy | Tumor Burden | Angiolipoma | Brain Neoplasms | Randomized Controlled Trials as Topic | Blood disorders | Child health | Medicine General & Introductory Medical Sciences | Administration, Topical | Everolimus | Astrocytoma | Seizures | Medicine, General & Internal | Life Sciences & Biomedicine | General & Internal Medicine | Science & Technology | Tuberous Sclerosis - complications | Humans | Immunosuppressive Agents - therapeutic use | Brain Neoplasms - pathology | Seizures - drug therapy | Male | Skin Diseases - pathology | Tuberous Sclerosis - pathology | Astrocytoma - pathology | Astrocytoma - drug therapy | Everolimus - adverse effects | Everolimus - therapeutic use | Female | Sirolimus - adverse effects | Sirolimus - therapeutic use | Angiolipoma - drug therapy | Skin Diseases - drug therapy | Brain Neoplasms - drug therapy | Angiolipoma - pathology | Tuberous Sclerosis - drug therapy | Tumor Burden - drug effects | Seizures - etiology | Immunosuppressive Agents - adverse effects | Kidney Neoplasms - pathology | Kidney Neoplasms - drug therapy | Index Medicus
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2. Full Text A Randomized Trial of Factor VIII and Neutralizing Antibodies in Hemophilia A
by Peyvandi, Flora and Mannucci, Pier M and Garagiola, Isabella and El-Beshlawy, Amal and Elalfy, Mohsen and Ramanan, Vijay and Eshghi, Peyman and Hanagavadi, Suresh and Varadarajan, Ramabadran and Karimi, Mehran and Manglani, Mamta V and Ross, Cecil and Young, Guy and Seth, Tulika and Apte, Shashikant and Nayak, Dinesh M and Santagostino, Elena and Mancuso, Maria Elisa and Sandoval Gonzalez, Adriana C and Mahlangu, Johnny N and Bonanad Boix, Santiago and Cerqueira, Monica and Ewing, Nadia P and Male, Christoph and Owaidah, Tarek and Soto Arellano, Veronica and Kobrinsky, Nathan L and Majumdar, Suvankar and Perez Garrido, Rosario and Sachdeva, Anupam and Simpson, Mindy and Thomas, Mathew and Zanon, Ezio and Antmen, Bulent and Kavakli, Kaan and Manco-Johnson, Marilyn J and Martinez, Monica and Marzouka, Esperanza and Mazzucconi, Maria G and Neme, Daniela and Palomo Bravo, Angeles and Paredes Aguilera, Rogelio and Prezotti, Alessandra and Schmitt, Klaus and Wicklund, Brian M and Zulfikar, Bulent and Rosendaal, Frits R
The New England journal of medicine, ISSN 0028-4793, 05/2016, Volume 374, Issue 21, pp. 2054 - 2064
Medicine, General & Internal | Life Sciences & Biomedicine | General & Internal Medicine | Science & Technology | Factor VIII - immunology | Humans | Isoantibodies - analysis | Middle Aged | Child, Preschool | Infant | Male | Injections, Subcutaneous - adverse effects | von Willebrand Factor - therapeutic use | Incidence | Dose-Response Relationship, Drug | Young Adult | Hemorrhage - etiology | Factor VIII - antagonists & inhibitors | Adult | Drug Therapy, Combination | Child | Factor VIII - therapeutic use | Hemophilia A - immunology | Proportional Hazards Models | Hemophilia A - complications | Adolescent | Aged | Hemophilia A - drug therapy | Antibodies, Neutralizing - blood | Viral antibodies | Care and treatment | Usage | Hemophilia | Antibodies | Clinical trials | Dosage and administration | Immunoglobulins | Inhibitor drugs | FDA approval | Regression analysis | Patients | Evidence-based medicine | Coagulation factors | Von Willebrand factor | Alloantibodies | Drug therapy | Health risk assessment | Index Medicus | Abridged Index Medicus
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3. Full Text Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy
by Pasi, K. John and Rangarajan, Savita and Georgiev, Pencho and Mant, Tim and Creagh, Michael D and Lissitchkov, Toshko and Bevan, David and Austin, Steve and Hay, Charles R and Hegemann, Inga and Kazmi, Rashid and Chowdary, Pratima and Gercheva-Kyuchukova, Liana and Mamonov, Vasily and Timofeeva, Margarita and Soh, Chang-Heok and Garg, Pushkal and Vaishnaw, Akshay and Akinc, Akin and Sørensen, Benny and Ragni, Margaret V
The New England journal of medicine, ISSN 0028-4793, 08/2017, Volume 377, Issue 9, pp. 819 - 828
Hemophilia B - therapy | Single-Blind Method | Humans | Middle Aged | Hemophilia B - blood | Male | Healthy Volunteers | Hemophilia A - therapy | Hemophilia A - blood | Dose-Response Relationship, Drug | Young Adult | Thrombin - biosynthesis | Antithrombin III - antagonists & inhibitors | Injections, Subcutaneous | Antithrombins - blood | Adult | RNAi Therapeutics | Care and treatment | Usage | Antithrombin III | Hemophilia | Hemophilia B | Research | Gene therapy | Plasma | Intravenous administration | Hematology | RNA-mediated interference | Liver | Body weight | Thrombin | Injection | Antithrombin | Clotting | Plasma levels | Alloantibodies | Thromboembolism | Drug dosages | Index Medicus | Abridged Index Medicus
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4. Full Text Rituximab in the treatment of autoimmune haematological disorders
by Garvey, Bernadette
British journal of haematology, ISSN 0007-1048, 04/2008, Volume 141, Issue 2, pp. 149 - 169
idiopathic thrombocytopenic purpura | thrombotic thrombocytopenic purpura | acquired haemophilia | cold agglutinin disease | autoimmune haemolytic anaemia | rituximab | Rituximab | Autoimmune haemolytic anaemia | Cold agglutinin disease | Idiopathic thrombocytopenic purpura | Thrombotic thrombocytopenic purpura | Acquired haemophilia | Life Sciences & Biomedicine | Hematology | Science & Technology | Hematologic and hematopoietic diseases | Anemias. Hemoglobinopathies | Biological and medical sciences | Medical sciences | Diseases of red blood cells | Platelet diseases and coagulopathies | Antigens, CD20 - immunology | Antibodies, Monoclonal, Murine-Derived | Humans | Middle Aged | Anemia, Hemolytic, Autoimmune - drug therapy | Hematologic Diseases - drug therapy | Antibodies, Monoclonal - therapeutic use | Purpura, Thrombocytopenic, Idiopathic - drug therapy | Aged, 80 and over | Purpura, Thrombotic Thrombocytopenic - drug therapy | Adult | Aged | Hemophilia A - drug therapy | Autoimmune Diseases - drug therapy | Drug therapy | Hemophilia | Index Medicus
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5. Full Text Pharmaceutical therapies to recode nonsense mutations in inherited diseases
by Lee, Hui-Ling Rose and Dougherty, Joseph P
Pharmacology & therapeutics (Oxford), ISSN 0163-7258, 11/2012, Volume 136, Issue 2, pp. 227 - 266
Inducible expression system | Premature termination codon | Nonsense suppression therapy | Aminoglycoside | Non-aminoglycoside | Readthrough | Nonsense codon | Life Sciences & Biomedicine | Pharmacology & Pharmacy | Science & Technology | Muscular Dystrophy, Duchenne - drug therapy | Humans | Gene Expression Regulation | Genetic Diseases, Inborn - genetics | Codon, Nonsense - drug effects | RNA, Messenger - analysis | Rett Syndrome - drug therapy | Animals | Cystic Fibrosis - genetics | Hemophilia A - genetics | Muscular Dystrophy, Duchenne - genetics | Hemophilia A - drug therapy | Rett Syndrome - genetics | Cystic Fibrosis - drug therapy | Genetic Diseases, Inborn - drug therapy | Aminoglycosides - therapeutic use | Inositol | Luciferase | Cystic fibrosis | Biosynthesis | Gene expression | Prothrombin complex concentrate | Carnitine | Blood coagulation factor VIII | Codon | Hemophilia | Physiological aspects | Genetic aspects | Pharmaceutical industry | Enzyme-linked immunosorbent assay | Protein binding | Index Medicus | Nonsense mutation | Duchenne's muscular dystrophy | Amino acids | spinal muscular atrophy | Gene therapy | Epidemiology | Cancer | Hereditary diseases | Pharmaceuticals
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6. Full Text 2017 Clinical trials update: Innovations in hemophilia therapy
by Hartmann, Jan and Croteau, Stacy E
American journal of hematology, ISSN 0361-8609, 12/2016, Volume 91, Issue 12, pp. 1252 - 1260
Life Sciences & Biomedicine | Hematology | Science & Technology | Blood Coagulation - drug effects | Factor IX - therapeutic use | Humans | Recombinant Fusion Proteins - therapeutic use | Half-Life | Clinical Trials as Topic | Hemophilia A - drug therapy | Factor VIII - therapeutic use | Genetic Therapy - methods | Hemophilia B - drug therapy | Proteins | Viral antibodies | Exercise | Blood coagulation factor VIII | Hemophilia | Clinical trials | Antibodies | Gene therapy | Patient compliance | Index Medicus
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