المؤلفون: Husain Attarwala, Valerie A. Clausen, Pushkal Garg, Varun Goel, Verena Karsten, Gabriel J. Robbie, Megan Melch, Akshay Vaishnaw, Bahru A. Habtemariam, John Vest, Marianne T. Sweetser

المصدر: Clinical Pharmacology & Therapeutics. 109:372-382

مصطلحات موضوعية: Adult, Male, Drug, Acetylgalactosamine, media_common.quotation_subject, Pharmacology, 030226 pharmacology & pharmacy, Drug Administration Schedule, N-Acetylgalactosamine, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Asian People, Pharmacokinetics, Humans, Prealbumin, Medicine, Single-Blind Method, Pharmacology (medical), Adverse effect, media_common, Amyloid Neuropathies, Familial, Dose-Response Relationship, Drug, biology, business.industry, Amyloidosis, medicine.disease, Healthy Volunteers, Transthyretin, chemistry, 030220 oncology & carcinogenesis, Pharmacodynamics, biology.protein, RNA, Female, business, Half-Life, Conjugate

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a926fdfa0bbe0b749b1e462a6eaad07dTest
https://doi.org/10.1002/cpt.1974Test

المؤلفون: Daniel P. Judge, Arnt V. Kristen, Martha Grogan, Mathew S. Maurer, Rodney H. Falk, Mazen Hanna, Julian Gillmore, Pushkal Garg, Akshay K. Vaishnaw, Jamie Harrop, Christine Powell, Verena Karsten, Xiaoping Zhang, Marianne T. Sweetser, John Vest, Philip N. Hawkins

المصدر: Cardiovascular Drugs and Therapy
Cardiovasc Drugs Ther

مصطلحات موضوعية: Adult, Male, Canada, Time Factors, Cardiomyopathy, 030204 cardiovascular system & hematology, Revusiran, 03 medical and health sciences, 0302 clinical medicine, RNA interference, Cause of Death, Humans, Prealbumin, ATTR amyloidosis, Pharmacology (medical), Genetic Predisposition to Disease, RNA, Small Interfering, Aged, Pharmacology, Aged, 80 and over, Amyloid Neuropathies, Familial, Exercise Tolerance, Correction, General Medicine, Recovery of Function, Middle Aged, United States, Europe, Phenotype, RNAi Therapeutics, Treatment Outcome, Early Termination of Clinical Trials, Mutation, Disease Progression, Female, Original Article, Cardiology and Cardiovascular Medicine, Cardiomyopathies, 030217 neurology & neurosurgery

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::45887ee13b195341e5d70adae8db7d93Test
http://europepmc.org/articles/PMC7242280Test

المؤلفون: Bruce Wang, Paolo Ventura, Kei-ichiro Takase, Manish Thapar, David Cassiman, Ilja Kubisch, Shangbin Liu, Marianne T. Sweetser, Manisha Balwani

المصدر: Orphanet Journal of Rare Diseases, vol 17, iss 1

مصطلحات موضوعية: Acute Porphyrias, Acute hepatic porphyria, Givosiran, Disease burden, Porphyria attack, Chronic symptoms, Quality of life, Porphobilinogen, Neurodegenerative, Research & Experimental Medicine, ACUTE INTERMITTENT PORPHYRIA, Hepatic, Cost of Illness, Envision, Pharmacology (medical), Genetics (clinical), Burden, Acute Porphyrias, Envision, Genetics & Heredity, Pain Research, PAIN, Hematology, Disease burden, General Medicine, LIVER-TRANSPLANTATION, Porphyria attack, Medicine, Research & Experimental, 6.1 Pharmaceuticals, Hemin, Chronic Pain, Life Sciences & Biomedicine, Quality of life, Clinical Trials and Supportive Activities, Pain, Burden, Porphyrias, Clinical Research, Acute hepatic porphyria, Genetics, MANAGEMENT, Humans, Peripheral Neuropathy, Givosiran, Other Medical and Health Sciences, Science & Technology, Porphyria, Chronic symptoms, Neurosciences, Evaluation of treatments and therapeutic interventions, Porphobilinogen Synthase, Acute Intermittent, Porphyrias, Hepatic, Porphyria, Acute Intermittent, Quality of Life, UPDATE, Digestive Diseases, ATTACKS

وصف الملف: Electronic; application/pdf

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d3dcc4a34a20afd30da2fd2112dd67d6Test
https://hdl.handle.net/11380/1261337Test

المؤلفون: Marianne T. Sweetser, Márcia Waddington-Cruz, Josep M. Campistol, Hartmut Schmidt, John L. Berk, Jing Jing Wang, Jared Gollob, David H. Adams, Teresa Coelho, Juan Buades, Michael Polydefkis, Jihong Chen, Ole B. Suhr, Isabel Conceição

المساهمون: Repositório da Universidade de Lisboa

المصدر: Orphanet Journal of Rare Diseases
Repositório Científico de Acesso Aberto de Portugal
Repositório Científico de Acesso Aberto de Portugal (RCAAP)
instacron:RCAAP
Orphanet Journal of Rare Diseases, Vol 15, Iss 1, Pp 1-10 (2020)

مصطلحات موضوعية: Tafamidis, medicine.medical_specialty, Neurologi, Cardiomyopathy, lcsh:Medicine, 030204 cardiovascular system & hematology, Gastroenterology, 03 medical and health sciences, chemistry.chemical_compound, RNA interference, 0302 clinical medicine, NA interference, Polyneuropathy, Internal medicine, medicine, Humans, Prealbumin, ATTR amyloidosis, Pharmacology (medical), RNA, Small Interfering, Adverse effect, Genetics (clinical), Amyloid Neuropathies, Familial, biology, business.industry, Research, Amyloidosis, lcsh:R, General Medicine, medicine.disease, Discontinuation, Transthyretin, Neurology, chemistry, Concomitant, Quality of Life, Patisiran, biology.protein, business, 030217 neurology & neurosurgery

وصف الملف: application/pdf

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::4ab76833c3c740a5a556579909bdfbdaTest
https://doi.org/10.1186/s13023-020-01399-4Test

المؤلفون: Marianne T. Sweetser, Gabriel J. Robbie, Xiaoping Zhang, Varun Goel, Valerie A. Clausen, Husain Attarwala

المصدر: Journal of Clinical Pharmacology

مصطلحات موضوعية: Male, patisiran, Therapeutics, Pharmacology, Placebo, 030226 pharmacology & pharmacy, small interfering ribonucleic acid (siRNA), 03 medical and health sciences, Polyneuropathies, 0302 clinical medicine, Pharmacokinetics, Double-Blind Method, pharmacokinetics (PK), Medicine, Humans, Prealbumin, Pharmacology (medical), Dosing, RNA, Small Interfering, Adverse effect, Aged, Amyloid Neuropathies, Familial, biology, Dose-Response Relationship, Drug, business.industry, Amyloidosis, Middle Aged, pharmacodynamics (PD), medicine.disease, Transthyretin, RNAi Therapeutics, Treatment Outcome, Liver, 030220 oncology & carcinogenesis, Pharmacodynamics, Liposomes, biology.protein, Nanoparticles, Administration, Intravenous, Female, exposure‐response, business, Polyneuropathy, hereditary transthyretin‐mediated amyloidosis (hATTR)

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9565a43aa8b1fa7cdcb2fe7d26f12920Test
https://pubmed.ncbi.nlm.nih.gov/31322739Test

المؤلفون: Xiaojun You, James G. Krueger, Firas Hougeir, Adam J. Friedman, Nisha Lucas, Marianne T. Sweetser, Jacob Elkins, Steven J. Greenberg, Wanda Castro-Borrero, Leon H Kircik, Peter McCroskery

المصدر: Advances in Therapy

مصطلحات موضوعية: Adult, Male, medicine.medical_specialty, Daclizumab, MedDRA, Phases of clinical research, Dermatology, Antibodies, Monoclonal, Humanized, Severity of Illness Index, Skin Diseases, Gastroenterology, 030207 dermatology & venereal diseases, 03 medical and health sciences, Multiple Sclerosis, Relapsing-Remitting, 0302 clinical medicine, Double-Blind Method, Internal medicine, Severity of illness, medicine, Humans, Pharmacology (medical), Adverse effect, Daclizumab high-yield process, Original Research, Cutaneous events, Medicine(all), business.industry, Incidence, Multiple sclerosis, Interferon beta-1a, General Medicine, Middle Aged, Interferon beta, medicine.disease, Rash, Treatment Outcome, Neurology, Immunoglobulin G, Immunology, Relapsing-remitting multiple sclerosis, Female, Safety, medicine.symptom, business, 030217 neurology & neurosurgery, medicine.drug

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a38992de9f4f237336503be280f49005Test
https://doi.org/10.1007/s12325-016-0353-2Test

المؤلفون: Mazen Hanna, Pushkal Garg, Arnt V. Kristen, Akshay Vaishnaw, Marianne T. Sweetser, Christine Powell, Verena Karsten, Julian D. Gillmore, Rodney H. Falk, Xiaoping Zhang, Mathew S. Maurer, Philip N. Hawkins, Daniel P. Judge, Jamie Harrop, John Vest, Martha Grogan

المصدر: Cardiovascular Drugs and Therapy. 34:889-889

مصطلحات موضوعية: 0301 basic medicine, medicine.medical_specialty, Cardiomyopathy, 030204 cardiovascular system & hematology, 03 medical and health sciences, 0302 clinical medicine, Internal medicine, Medicine, Pharmacology (medical), In patient, Interventricular septum, Pharmacology, biology, business.industry, Amyloidosis, General Medicine, medicine.disease, Transthyretin, 030104 developmental biology, medicine.anatomical_structure, Multicenter study, biology.protein, Cardiology, Cardiology and Cardiovascular Medicine, business, Wall thickness, Attr amyloidosis

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::e0bc0bd56844fc2433386c22184287a6Test
https://doi.org/10.1007/s10557-020-07023-8Test

المؤلفون: Eva Havrdova, Michael Hutchinson, Ralf Gold, Marianne T. Sweetser, Katherine Dawson, Nuwan Kurukulasuriya, Kartik Raghupathi

المصدر: Expert Opinion on Pharmacotherapy. 14:2145-2156

مصطلحات موضوعية: Male, Oncology, medicine.medical_specialty, Disease, Pharmacology, law.invention, chemistry.chemical_compound, Multiple Sclerosis, Relapsing-Remitting, Fumarates, Randomized controlled trial, law, Internal medicine, Humans, Medicine, Pharmacology (medical), Adverse effect, Dimethyl fumarate, business.industry, Multiple sclerosis, Incidence (epidemiology), General Medicine, medicine.disease, Relapsing remitting, chemistry, Expert opinion, Female, Peptides, business, Immunosuppressive Agents

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::feaa7f03888a2d998ac81c6f8b666c5dTest
https://doi.org/10.1517/14656566.2013.826190Test

المؤلفون: Marianne T. Sweetser, Kye Gilder, Jeffrey L. Kaine, Maria Greenwald, Matthew D. Linnik, William Shergy

المصدر: Arthritis & Rheumatism. 63:622-632

مصطلحات موضوعية: Adult, Male, medicine.medical_specialty, Adolescent, medicine.medical_treatment, Immunology, Arthritis, Antibodies, Monoclonal, Humanized, Placebo, Gastroenterology, Receptors, Tumor Necrosis Factor, Etanercept, Arthritis, Rheumatoid, Placebos, Antibodies, Monoclonal, Murine-Derived, Young Adult, Double-Blind Method, Rheumatology, immune system diseases, Internal medicine, medicine, Adalimumab, Humans, Immunology and Allergy, Pharmacology (medical), Infusions, Intravenous, Aged, Tumor Necrosis Factor-alpha, business.industry, Middle Aged, medicine.disease, TNF inhibitor, Surgery, Methotrexate, Antirheumatic Agents, Immunoglobulin G, Rheumatoid arthritis, Acute Disease, Drug Therapy, Combination, Female, Rituximab, business, medicine.drug

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::65fb1a7344eace7169330615794eeb89Test
https://doi.org/10.1002/art.30194Test

المؤلفون: Gerald G. Krueger, Mohamed Darif, Kim A. Papp, Bernard S. Goffe, Marianne T. Sweetser, Barry Ticho, Carmen Bozic, Sophia Lee, David Gratton

المصدر: Clinical Therapeutics. 27:1912-1921

مصطلحات موضوعية: Adult, Male, medicine.medical_specialty, Adolescent, Recombinant Fusion Proteins, Population, Alefacept, Infections, Antibodies, Neoplasms, Psoriasis, Internal medicine, medicine, Humans, Pharmacology (medical), education, Adverse effect, Aged, Aged, 80 and over, Pharmacology, Clinical Trials as Topic, education.field_of_study, business.industry, Incidence (epidemiology), Middle Aged, medicine.disease, Surgery, Clinical trial, Upper respiratory tract infection, Tolerability, Female, Dermatologic Agents, business, medicine.drug

الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::e1bbb30b00e2253ff2b5844dfd3de039Test
https://doi.org/10.1016/j.clinthera.2005.12.007Test