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1Academic Journal
المؤلفون: Rachael A. Potter, Ida H. Moeller, Sohrab Khan, Hélène Haegel, Andreas Hollenstein, Guido Steiner, Christoph Wandel, Alexander P. Murphy, Damon R. Asher, Emanuel Palatinsky, Danielle A. Griffin, Stefanie Mason, Susan T. Iannaccone, Craig M. Zaidman, Louise R. Rodino-Klapac
المصدر: Scientific Reports, Vol 15, Iss 1, Pp 1-11 (2025)
مصطلحات موضوعية: AAV vector, Delandistrogene moxeparvovec, Duchenne muscular dystrophy, Dystrophin, Gene transfer therapy, Immune-mediated myositis, Medicine, Science
وصف الملف: electronic resource
Relation: https://doaj.org/toc/2045-2322
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2Academic Journal
المؤلفون: Aravindhan Veerapandiyan, Anne M. Connolly, Katherine D. Mathews, Stanley Nelson, Craig McDonald, Richard S. Finkel, Vettaikorumakankav Vedanarayanan, Cuixia Tian, Susan Apkon, Julie A. Parsons, Jonathan H. Soslow, William Bryan Burnette, Kaitlin Y. Batley, Susan T. Iannaccone, Carolina Tesi Rocha, Kevin M. Flanigan, Diana Bharucha‐Goebel, Sarah Wright, Migvis Monduy, Simona Treidler, Ashutosh Kumar, Nancy L. Kuntz, Vamshi K. Rao, Rachel Schrader, Saunder M. Bernes, Vikki Ann Stefans, Jena M. Krueger, Marcia V. Felker, Omer Abdul Hamid, Arpita Lakhotia, Susan Matesanz, Partha S. Ghosh, Natalie Katz, Hoda Abdel‐Hamid, Chamindra G. Laverty, Bo Hoon Lee, Amy Harper, Leigh Ramos‐Platt, Diana Castro, Russell J. Butterfield, Crystal M. Proud, Craig M. Zaidman, Emma Ciafaloni
المصدر: Annals of the Child Neurology Society, Vol 2, Iss 3, Pp 184-188 (2024)
مصطلحات موضوعية: Becker muscular dystrophy, DMD, Duchenne, dystrophinopathy, treatment, Neurology. Diseases of the nervous system, RC346-429, Pediatrics, RJ1-570
وصف الملف: electronic resource
Relation: https://doaj.org/toc/2831-3267
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3Academic Journal
المصدر: Бюллетень сибирской медицины, Vol 9, Iss 6, Pp 34-40 (2010)
مصطلحات موضوعية: нейрофиброматоз, пластинка роста, склеротом, деформация позвоночника, экспрессия гена, Medicine
وصف الملف: electronic resource
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4Academic Journal
المؤلفون: E. L. Strokova, N. Yu. Pakhomova, A. I. Shevchenko, A. A. Korytkin, V. V. Kozhevnikov, А. M. Zaidman
المصدر: Сибирский научный медицинский журнал, Vol 43, Iss 6, Pp 203-209 (2024)
مصطلحات موضوعية: funnel-shaped chest deformity, cell culture, Medicine
وصف الملف: electronic resource
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5Academic Journal
المصدر: International Journal of Neonatal Screening, Vol 10, Iss 3, p 58 (2024)
مصطلحات موضوعية: spinal muscular atrophy, newborn screening, treatment practices, birth prevalence, gene therapy, Pediatrics, RJ1-570
وصف الملف: electronic resource
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6Academic Journal
المؤلفون: Natalie L. Goedeker, Sachi D. Dharia, Danielle A. Griffin, Jesantha Coy, Todd Truesdale, Rajan Parikh, Kasen Whitehouse, Sourav Santra, Damon R. Asher, Craig M. Zaidman
المصدر: Therapeutic Advances in Neurological Disorders, Vol 16 (2023)
مصطلحات موضوعية: Neurology. Diseases of the nervous system, RC346-429
Relation: https://doi.org/10.1177/17562864221149781; https://doaj.org/toc/1756-2864; https://doaj.org/article/3188b9979c4a41868a6b7e86441ae1d1
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7
المؤلفون: Anne M. Connolly, Craig M. Zaidman, John F. Brandsema, Han C. Phan, Cuixia Tian, Xueping Zhang, Jack Li, Mark D. Eisner, Ewa Carrier
المصدر: Journal of Neuromuscular Diseases. :1-15
مصطلحات موضوعية: Neurology, Neurology (clinical)
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8
المؤلفون: Richard S. Finkel, Basil T. Darras, Jerry R. Mendell, John W. Day, Nancy L. Kuntz, Anne M. Connolly, Craig M. Zaidman, Thomas O. Crawford, Russell J. Butterfield, Perry B. Shieh, Gihan Tennekoon, John F. Brandsema, Susan T. Iannaccone, John Shoffner, Sarah Kavanagh, Thomas A. Macek, Sitra Tauscher-Wisniewski
المصدر: Journal of Neuromuscular Diseases. 10:389-404
مصطلحات موضوعية: Neurology, Neurology (clinical)
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9Academic Journal
المؤلفون: Sarah R Rivera, Sumit K Jhamb, Hoda Z Abdel-Hamid, Gyula Acsadi, John Brandsema, Emma Ciafaloni, Basil T Darras, Susan T Iannaccone, Chamindra G Konersman, Nancy L Kuntz, Craig M McDonald, Julie A Parsons, Carolina Tesi Rocha, Craig M Zaidman, Russell J Butterfield, Anne M Connolly, Katherine D Mathews
المصدر: PLoS ONE, Vol 15, Iss 10, p e0240687 (2020)
وصف الملف: electronic resource
Relation: https://doaj.org/toc/1932-6203
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10Academic Journal
المؤلفون: Alla M. Zaidman
المصدر: Хирургия позвоночника, Vol 17, Iss 3, Pp 117-133 (2020)
مصطلحات موضوعية: idiopathic scoliosis, vertebral body growth plates, proteoglycans, scoliosis model, Surgery, RD1-811
Relation: https://www.spinesurgery.ru/jour/article/view/1731/1719; https://doaj.org/toc/1810-8997; https://doaj.org/toc/2313-1497; https://doaj.org/article/703499ac283942c0837bbc18bf8bf020
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11
المؤلفون: Kevin A. Strauss, Michelle A. Farrar, Francesco Muntoni, Kayoko Saito, Jerry R. Mendell, Laurent Servais, Hugh J. McMillan, Richard S. Finkel, Kathryn J. Swoboda, Jennifer M. Kwon, Craig M. Zaidman, Claudia A. Chiriboga, Susan T. Iannaccone, Jena M. Krueger, Julie A. Parsons, Perry B. Shieh, Sarah Kavanagh, Sitra Tauscher-Wisniewski, Bryan E. McGill, Thomas A. Macek
المصدر: Nature Medicine. 28:1381-1389
مصطلحات موضوعية: General Medicine, General Biochemistry, Genetics and Molecular Biology
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12
المؤلفون: Kevin A. Strauss, Michelle A. Farrar, Francesco Muntoni, Kayoko Saito, Jerry R. Mendell, Laurent Servais, Hugh J. McMillan, Richard S. Finkel, Kathryn J. Swoboda, Jennifer M. Kwon, Craig M. Zaidman, Claudia A. Chiriboga, Susan T. Iannaccone, Jena M. Krueger, Julie A. Parsons, Perry B. Shieh, Sarah Kavanagh, Melissa Wigderson, Sitra Tauscher-Wisniewski, Bryan E. McGill, Thomas A. Macek
المصدر: Nature Medicine. 28:1390-1397
مصطلحات موضوعية: General Medicine, General Biochemistry, Genetics and Molecular Biology
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13
المؤلفون: Megan A. Waldrop, Steven A. Moore, Katherine D. Mathews, Benjamin W. Darbro, Livja Medne, Richard Finkel, Anne M. Connolly, Thomas O. Crawford, Daniel Drachman, Nicolas Wein, Ali A. Habib, Monika A. Krzesniak‐Swinarska, Craig M. Zaidman, James J. Collins, Manu Jokela, Bjarne Udd, John W. Day, Gloria Ortiz‐Guerrero, Jeff Statland, Russell J. Butterfield, Diane M. Dunn, Robert B. Weiss, Kevin M. Flanigan
المصدر: Hum Mutat
Human mutation, vol 43, iss 4مصطلحات موضوعية: Duchenne muscular dystrophy, Duchenne/ Becker Muscular Dystrophy, pseudoexon, deep intronic, Intellectual and Developmental Disabilities (IDD), Clinical Sciences, Article, Dystrophin, Rare Diseases, Genetics, Humans, Muscular Dystrophy, Genetics (clinical), transcription termination, Pediatric, Genetics & Heredity, Human Genome, telescripting, Duchenne, Introns, Brain Disorders, Muscular Dystrophy, Duchenne, Becker muscular dystrophy, Musculoskeletal, Mutation, RNA Splice Sites, Biotechnology
وصف الملف: application/pdf
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14Academic Journal
المؤلفون: Ryan W Johnson, Kay W P Ng, Alexander R Dietz, Mary E Hartman, Jack D Baty, Nausheen Hasan, Craig M Zaidman, Michael Shoykhet
المصدر: PLoS ONE, Vol 13, Iss 12, p e0207720 (2018)
وصف الملف: electronic resource
Relation: https://doaj.org/toc/1932-6203
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15Academic Journal
المؤلفون: Alla M. Zaidman
المصدر: Хирургия позвоночника, Vol 15, Iss 2, Pp 91-97 (2018)
مصطلحات موضوعية: bone tissue, chondro-osseous graft, osteogenesis, Surgery, RD1-811
Relation: https://www.spinesurgery.ru/jour/article/view/1474/1416; https://doaj.org/toc/1810-8997; https://doaj.org/toc/2313-1497; https://doaj.org/article/6c7b32a2500b497e998e44c436e705b7
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16Academic Journal
المؤلفون: Victor V. Rerikh, Yury A. Predein, Alla M. Zaidman, Alexey D. Lastevsky, Vladimir A. Bataev, Aelita A. Nikulina
المصدر: Хирургия позвоночника, Vol 15, Iss 4, Pp 41-51 (2018)
مصطلحات موضوعية: allogeneic bone graft, vertebral body plasty, vertebral fracture, autobone, microhardness, mineralization, spectrometry, osteogenesis, Surgery, RD1-811
Relation: https://www.spinesurgery.ru/jour/article/view/1515/1490; https://doaj.org/toc/1810-8997; https://doaj.org/toc/2313-1497; https://doaj.org/article/605dc85f599f4cdb80f8980ca88a0fce
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17
المؤلفون: Kapil Arya, Deepa H. Chand, Vikas R. Dharnidharka, Michelle A. Farrar, Sandra P. Reyna, Natalie L. Goedeker, Raja Dandamudi, Fiona E. Mackie, Craig M. Zaidman, Rachel Millner
المصدر: The Journal of Pediatrics. 231:265-268
مصطلحات موضوعية: Biological Products, Pathology, medicine.medical_specialty, Thrombotic microangiopathy, Thrombotic Microangiopathies, business.industry, Recombinant Fusion Proteins, Genetic enhancement, Infant, Spinal muscular atrophy, Motor neuron, medicine.disease, Viral vector, Muscular Atrophy, Spinal, 03 medical and health sciences, 0302 clinical medicine, medicine.anatomical_structure, 030225 pediatrics, Pediatrics, Perinatology and Child Health, medicine, Humans, Female, 030212 general & internal medicine, business
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18
المؤلفون: Craig M. Zaidman, Arun S. Varadhachary, Anne M. Connolly, Jennifer L Gibbons, Natalie L. Goedeker
المصدر: Muscle & Nerve. 63:902-905
مصطلحات موضوعية: Adult, Male, 0301 basic medicine, Adolescent, Physiology, Sedation, medicine.medical_treatment, Oligonucleotides, 030105 genetics & heredity, Asymptomatic, Muscular Atrophy, Spinal, Young Adult, 03 medical and health sciences, Cellular and Molecular Neuroscience, 0302 clinical medicine, Physiology (medical), medicine, Humans, Intubation, Child, Adverse effect, Injections, Spinal, Retrospective Studies, Intracranial pressure, business.industry, Infant, Retrospective cohort study, Middle Aged, Laboratories, Hospital, SMA, Child, Preschool, Anesthesia, Female, Nusinersen, Neurology (clinical), medicine.symptom, business, 030217 neurology & neurosurgery
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19Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy
المؤلفون: Paula R, Clemens, Vamshi K, Rao, Anne M, Connolly, Amy D, Harper, Jean K, Mah, Craig M, McDonald, Edward C, Smith, Craig M, Zaidman, Tomoyuki, Nakagawa, Eric P, Hoffman
المصدر: Journal of neuromuscular diseases. 9(4)
مصطلحات موضوعية: Dystrophin, Male, Muscular Dystrophy, Duchenne, Oligonucleotides, Humans, Oligonucleotides, Antisense
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20
المؤلفون: Amanda B. Rogers, Craig M. Zaidman, Anne M. Connolly
المصدر: Muscle & Nerve. 62:705-709
مصطلحات موضوعية: Male, 0301 basic medicine, Pediatrics, medicine.medical_specialty, Adolescent, Physiology, medicine.drug_class, Administration, Oral, Chronic inflammatory demyelinating polyneuropathy, 030105 genetics & heredity, Weight Gain, Maintenance Chemotherapy, Disease course, Young Adult, 03 medical and health sciences, Cellular and Molecular Neuroscience, 0302 clinical medicine, Maintenance therapy, Adrenal Cortex Hormones, Sleep Initiation and Maintenance Disorders, Physiology (medical), medicine, Humans, Immunologic Factors, Child, Glucocorticoids, Duration of Therapy, business.industry, Pulse (signal processing), Medical record, Immunoglobulins, Intravenous, Infant, Treatment options, medicine.disease, Irritable Mood, Polyradiculoneuropathy, Chronic Inflammatory Demyelinating, Pulse Therapy, Drug, Child, Preschool, Prednisone, Corticosteroid, Female, Neurology (clinical), business, Off Treatment, 030217 neurology & neurosurgery