يعرض 1 - 10 نتائج من 12 نتيجة بحث عن '"Raheel Shafi"', وقت الاستعلام: 1.55s تنقيح النتائج
  1. 1
    Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naive and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study

    المؤلفون: Philip Van Damme, Pascal Laforêt, Benedikt Schoser, Barry J. Byrne, Loren D.M. Pena, Richard J. Barohn, Ozlem Goker-Alpan, Kerry Culm-Merdek, Ans T. van der Ploeg, Volker Straub, Karl Eugen Mengel, Beth L. Thurberg, Jean Pouget, Raheel Shafi, Katherine Kacena, Alan Pestronk, Peter Young, John Vissing, Shafeeq Ladha, Claude Desnuelle, Jaya Trivedi

    المساهمون: Pediatrics

    المصدر: Neuromuscular Disorders, 29(3), 167-186. Elsevier Ltd.
    NEO1 Investigator Group 2019, ' Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease : A phase 1, open-label, multicenter, multinational, ascending dose study ', Neuromuscular Disorders, vol. 29, no. 3, pp. 167-186 . https://doi.org/10.1016/j.nmd.2018.12.004Test

    مصطلحات موضوعية: Avalglucosidase alfa (neoGAA), 0301 basic medicine, Male, GLUCOSE TETRASACCHARIDE, Lysosomal acid alpha-glucosidase (GAA) deficiency, CHILDREN, Pulmonary function testing, MOTOR FUNCTION, 0302 clinical medicine, Medicine, Genetics (clinical), Late-onset Pompe disease (LOPD), Glycogen Storage Disease Type II, Alglucosidase alfa, MOUSE MODEL, Enzyme replacement therapy, Middle Aged, Treatment Outcome, Neurology, Tolerability, SKELETAL-MUSCLE, Female, Life Sciences & Biomedicine, MUSCLE TRAINING RMT, Glycogen, 6-MINUTE WALK, medicine.drug, Adult, medicine.medical_specialty, Clinical Neurology, GLYCOGEN, 03 medical and health sciences, FEV1/FVC ratio, Pharmacokinetics, Internal medicine, Humans, Enzyme Replacement Therapy, Adverse effect, Science & Technology, business.industry, Neurosciences, alpha-Glucosidases, ADULTS, Glycogen storage disease type II, SEVERITY, 030104 developmental biology, Pharmacodynamics, Pediatrics, Perinatology and Child Health, Neurosciences & Neurology, Neurology (clinical), Glucan 1,4-alpha-Glucosidase, business, 030217 neurology & neurosurgery

    وصف الملف: Print-Electronic; application/pdf

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::cf2f385d766335b6e27ce068b0b3a3b8Test
    https://doi.org/10.1016/j.nmd.2018.12.004Test


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  2. 2
    Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study

    المؤلفون: Pranoot Tanpaiboon, Michael J. Gambello, Loren D.M. Pena, John W. Day, Raymond Y. Wang, David Kronn, James B. Gibson, Raheel Shafi, Catherine Wilson, Priya S. Kishnani, Kristina An Haack, Si Houn Hahn, David W. Stockton, Yang Zhao, Richard Hillman, Susan Sparks, Nancy D. Leslie

    المصدر: Genetics in Medicine. 20:1284-1294

    مصطلحات موضوعية: 0301 basic medicine, Vital capacity, medicine.medical_specialty, business.industry, Immunogenicity, Cardiomyopathy, Liter, Disease, medicine.disease, 03 medical and health sciences, Safety profile, 030104 developmental biology, 0302 clinical medicine, Internal medicine, medicine, Prospective cohort study, business, Alglucosidase alfa, 030217 neurology & neurosurgery, Genetics (clinical), medicine.drug

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::7d4135cde37504190e52c09116950560Test
    https://doi.org/10.1038/gim.2018.2Test


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  3. 3
    Efficacy, safety profile, and immunogenicity of alglucosidase alfa produced at the 4,000-liter scale in US children and adolescents with Pompe disease: ADVANCE, a phase IV, open-label, prospective study

    المؤلفون: Si Houn, Hahn, David, Kronn, Nancy D, Leslie, Loren D M, Pena, Pranoot, Tanpaiboon, Michael J, Gambello, James B, Gibson, Richard, Hillman, David W, Stockton, John W, Day, Raymond Y, Wang, Kristina, An Haack, Raheel, Shafi, Susan, Sparks, Yang, Zhao, Catherine, Wilson, Priya S, Kishnani

    المصدر: Genetics in medicine : official journal of the American College of Medical Genetics. 20(10)

    مصطلحات موضوعية: Male, Dose-Response Relationship, Drug, Glycogen Storage Disease Type II, Child, Preschool, Infant, Newborn, Humans, Infant, Female, alpha-Glucosidases, Kaplan-Meier Estimate, Age of Onset, Child, Recombinant Proteins

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=pmid________::da28f3c7274d5a7a7d72af25a662bf13Test
    https://pubmed.ncbi.nlm.nih.gov/29565424Test


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  4. 4
    Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study

    المؤلفون: Matthias Boentert, Volker Straub, Loren D.M. Pena, Peter Young, Beth L. Thurberg, Ans T. van der Ploeg, Robert Carlier, Alan Pestronk, Stephan Wenninger, Richard J. Barohn, Ozlem Goker-Alpan, Stephan C.A. Wens, Benedikt Schoser, Zachary Simmons, Carl Bjartmar, Michela Guglieri, Mazen M. Dimachkie, Raheel Shafi, John T. Kissel, Pierre Yves Baudin, Pierre G. Carlier, Tahseen Mozaffar

    المساهمون: Pediatrics, Neurology

    المصدر: Molecular Genetics and Metabolism, 119(1-2), 115-123. Academic Press

    مصطلحات موضوعية: 0301 basic medicine, Muscle tissue, Adult, Male, medicine.medical_specialty, Pathology, Endocrinology, Diabetes and Metabolism, Biopsy, Population, Urology, Biochemistry, 03 medical and health sciences, chemistry.chemical_compound, 0302 clinical medicine, Endocrinology, medicine, Genetics, Humans, Respiratory function, Enzyme Replacement Therapy, Age of Onset, education, Muscle, Skeletal, Alglucosidase alfa, Molecular Biology, Physical Therapy Modalities, Aged, education.field_of_study, Muscle biopsy, medicine.diagnostic_test, Glycogen, business.industry, Glycogen Storage Disease Type II, Skeletal muscle, alpha-Glucosidases, Enzyme replacement therapy, Middle Aged, Magnetic Resonance Imaging, 030104 developmental biology, medicine.anatomical_structure, Treatment Outcome, chemistry, Female, business, 030217 neurology & neurosurgery, medicine.drug

    وصف الملف: application/pdf

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::83937b970bd15b4ccc05645fa7bc06d2Test
    https://pure.eur.nl/en/publications/e5f667fe-293b-4998-9720-49d47c2c2492Test


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  5. 5
    Phase 1 safety and pharmacokinetics of the novel enzyme replacement therapy neoGAA in treatment-naïve and alglucosidase alfa-treated late-onset Pompe disease patients

    المؤلفون: Benedikt Schoser, Philip Van Damme, John Vissing, Kerry Culm-Merdek, Pascal Laforêt, Volker Straub, Shafeeq Ladha, Jaya Trivedi, Loren Pena, Eugen Mengel, Jean Pouget, Peter Young, Raheel Shafi, Alan Pestronk, Gerard Short

    المصدر: Molecular Genetics and Metabolism. 117:S117-S118

    مصطلحات موضوعية: medicine.medical_specialty, business.industry, Endocrinology, Diabetes and Metabolism, Late onset, Disease, Enzyme replacement therapy, Biochemistry, Gastroenterology, Therapy naive, Endocrinology, Pharmacokinetics, Internal medicine, Genetics, medicine, business, Molecular Biology, Alglucosidase alfa, medicine.drug

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::801b7f4a80c6fd4da29646e23764df03Test
    https://doi.org/10.1016/j.ymgme.2015.12.476Test


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  6. 6
    Heteropoly acid precursor to a catalyst for dibenzothiophene hydrodesulfurization

    المؤلفون: Raheel Shafi, Ivan V. Kozhevnikov, M. Rafiq H. Siddiqui, Eric G. Derouane, Graham J. Hutchings

    المصدر: Applied Catalysis A: General. 204:251-256

    مصطلحات موضوعية: Thermogravimetric analysis, chemistry.chemical_compound, Chemistry, Dibenzothiophene, Process Chemistry and Technology, Yield (chemistry), Inorganic chemistry, Trickle-bed reactor, Heterogeneous catalysis, Hydrodesulfurization, Catalysis, Space velocity

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_________::4d12f8f7610df38520c807f12b5e7fecTest
    https://doi.org/10.1016/s0926-860xTest(00)00530-5


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  7. 7
    A phase 4 prospective study in patients with adult Pompe disease treated with alglucosidase alfa

    المؤلفون: Tahseen Mozaffar, Carl Bjartmar, Volker Straub, John T. Kissel, Ans T. van der Ploeg, Zachary Simmons, Richard J. Barohn, Beth L. Thurberg, Loren D.M. Pena, Ozlem Goker-Alpan, Alan Pestronk, Pierre G. Carlier, Peter Young, Raheel Shafi, Benedikt Schoser

    المصدر: Molecular Genetics and Metabolism. 114:S113-S114

    مصطلحات موضوعية: Pediatrics, medicine.medical_specialty, business.industry, Endocrinology, Diabetes and Metabolism, Disease, Biochemistry, Endocrinology, Neurology, Genetics, Medicine, In patient, Neurology (clinical), business, Prospective cohort study, Molecular Biology, Alglucosidase alfa, medicine.drug

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::21d8e18205776a5e387f2feb08b2f4b1Test
    https://doi.org/10.1016/j.ymgme.2014.12.259Test


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  8. 8
    Distal Femoral Osteotomy: Is Internal Fixation Better than External?

    المؤلفون: Raheel Shafi, Austin T. Fragomen, K. T. Matthew Seah, S. Robert Rozbruch

    مصطلحات موضوعية: Adult, Male, medicine.medical_specialty, Time Factors, External Fixators, Knee Joint, medicine.medical_treatment, Bone healing, Osteotomy, External fixation, Fracture Fixation, Internal, Intraoperative Period, Postoperative Complications, Clinical Research, medicine, Internal fixation, Humans, Orthopedics and Sports Medicine, Femur, Range of Motion, Articular, Retrospective Studies, Fracture Healing, Osteosynthesis, business.industry, fungi, food and beverages, General Medicine, Recovery of Function, Surgery, Joint Deformities, Acquired, Orthopedic surgery, Female, business

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6d716511b428a59443eae79c27383786Test
    https://europepmc.org/articles/PMC3111789Test/


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  9. 9
    Ipsilateral Fibular Transport Using Ilizarov–Taylor Spatial Frame for a Limb Salvage Reconstruction: a Case Report

    المؤلفون: Austin T. Fragomen, S. Robert Rozbruch, Raheel Shafi

    مصطلحات موضوعية: medicine.medical_specialty, business.industry, Limb salvage, Soft tissue, medicine.disease, musculoskeletal system, Article, Surgery, Orthopedic surgery, Soft tissue injury, Taylor Spatial Frame, medicine, Orthopedics and Sports Medicine, Tibia, business

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::aef410ac3e69f97cc957faef6e3baa7dTest
    https://europepmc.org/articles/PMC2642540Test/


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  10. 10
    Treatment of dynamic scaphoid instability

    المؤلفون: Carrie Baer, Winfred Parker, R A Raheel Shafi, Houshang Seradge, David Dalsimer, Ali Seradge

    المصدر: The Journal of trauma. 56(6)

    مصطلحات موضوعية: Joint Instability, Wrist Joint, medicine.medical_specialty, Tendon Transfer, Wrist pain, Wrist, Critical Care and Intensive Care Medicine, Grip strength, Arthroscopy, Hand strength, medicine, Deformity, Humans, Rupture, Scaphoid Bone, medicine.diagnostic_test, Hand Strength, business.industry, Surgery, Biomechanical Phenomena, body regions, Radiography, medicine.anatomical_structure, Orthopedic surgery, Ligaments, Articular, Extensor Carpi Radialis Longus, medicine.symptom, business

    الوصول الحر: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a7025a0f371c9858af2498437840a9a4Test
    https://pubmed.ncbi.nlm.nih.gov/15211134Test


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