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Your search keyword '"Jerry R. Mendell"' showing total 7 results
Start Over Author "Jerry R. Mendell" Publisher georg thieme verlag kg
7 results on '"Jerry R. Mendell"'

1. Safety, β-Sarcoglycan Expression, and Functional Outcomes from Systemic Gene Transfer of rAAVrh74.MHCK7.hSGCB in LGMD2E/R4

Author

Jerry R. Mendell, E. R. Pozsgai, Linda Lowes, S. Neuhaus, M. A. Iammarino, A. S. Meadows, Kelly J. Lehman, Danielle A. Griffin, N. F. Reash, E. Koenig, A. Mueller-York, Sarah Lewis, L. R. Rodino-Klapac, X. Li, and Kathleen Church

Subjects
Sarcoglycan, Expression (architecture), business.industry, Medicine, Gene transfer, business, Cell biology
Published
2021
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2. Systemic Gene Transfer with rAAVrh74.MHCK7.SGCB Increased -sarcoglycan Expression in Patients with Limb Girdle Muscular Dystrophy Type 2E

Author

N. Miller, Kathleen Church, Eric R. Pozsgai, Danielle A. Griffin, Aaron Meadows, Sarah Lewis, Jerry R. Mendell, Louise R. Rodino-Klapac, Linda Lowes, Kelly J. Lehman, and M. A. Iammarino

Subjects
Pathology, medicine.medical_specialty, Sarcoglycan, business.industry, Medicine, Gene transfer, In patient, business, medicine.disease, Limb-girdle muscular dystrophy
Published
2019
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3. Genersatztherapie (Gene Replacement Therapy, GRT) mit AVXS-101 bei spinaler Muskelatrophie Typ I (SMA1): Pivotstudie (STR1VE) – Aktualisierung

Author

Lynlee M Muehring, Anne M. Connolly, Douglas M. Sproule, James L’Italien, Claudia A. Chiriboga, Susan T. Iannaccone, L.D.M. Peña, Thomas O. Crawford, John W. Day, Thomas A. Macek, Douglas E. Feltner, Brian K. Kaspar, Richard S. Finkel, Perry B. Shieh, Nancy L. Kuntz, Edward C. Smith, Basil T. Darras, S Nagendran, Meredith Schultz, Jerry R. Mendell, Elaine Kernbauer, and Francis G. Ogrinc

Published
2019
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4. Clinical Outcome Study for Dysferlinopathy: Three years of natural history data for clinical trial readiness

Author

Anna Mayhew, K. Bushby, M. James, John W. Day, T Stojkovic, Carmen Paradas, Maggie C. Walter, Sabine Krause, Alan Pestronk, Volker Straub, M. Yoshimura, Kristi J. Jones, Diana Bharucha-Goebel, J. Diaz-Manera, Elena Bravver, Simone Spuler, E Salort Campana, Jerry R. Mendell, and Elena Pegoraro

Subjects
Natural history, Clinical trial, Dysferlinopathy, medicine.medical_specialty, business.industry, Physical therapy, Medicine, business, medicine.disease, Outcome (game theory)
Published
2019
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5. Clinical Outcome Study in Dysferlinopathy: Medical comorbidities and polytherapy in a large population of dysferlinopathy patients

Author

Sabine Krause, E Salort Campana, K Storch, John W. Day, Kristi J. Jones, Elena Bravver, Maggie C. Walter, Jerry R. Mendell, V. Straub, J.D. Manera, Elena Pegoraro, K. Bushby, Alan Pestronk, Madoka Mori-Yoshimura, Carmen Paradas, T. Stojkovic, Simone Spuler, and Diana Bharucha-Goebel

Subjects
Pediatrics, medicine.medical_specialty, Dysferlinopathy, business.industry, Large population, medicine, business, medicine.disease, Outcome (game theory)
Published
2019
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6. IP 853. AVXS-101 Phase-1-Gene Therapy Clinical Trial in SMA Type 1: Event-Free Survival and Achievement of Developmental Milestones

Author

Kathrin Meyer, Arthur H.M. Burghes, James L’Italien, Linda Lowes, Kathleen Church, Samiah Al-Zaidy, Richard Shell, Thomas W. Prior, Courtney Wells, William Arnold, Brian K. Kaspar, Sukumar Nagendran, Louise R. Rodino-Klapac, Jerry R. Mendell, Foust Kevin, Shibi Likhite, John T. Kissel, K. Berry, Lindsay N. Alfano, and Douglas M. Sproule

Subjects
Clinical trial, Oncology, medicine.medical_specialty, business.industry, Internal medicine, Genetic enhancement, Event free survival, Developmental Milestone, medicine, SMA, business
Published
2018
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7. Muscular Dystrophy: Historical Overview and Classification in the Genetic Era

Author

John T. Kissel and Jerry R. Mendell

Subjects
Cognitive science, Duchenne dystrophy, business.industry, Classification, medicine.disease, Myotonic dystrophy, Muscular Dystrophies, Muscular Diseases, Neurology, Mutation, medicine, Humans, Classification methods, Neurology (clinical), Muscular dystrophy, medicine.symptom, business, Molecular Biology, Confusion
Abstract

Despite recent advances in molecular genetics, it has proven very difficult to arrive at an accurate and clinically useful classification of the muscular dystrophies. Much of this difficulty arises from confusion related to the term "muscular dystrophy" itself, as well as a general reluctance on the part of the neuromuscular community to abandon traditional, clinically based classifications. Nevertheless, advances in the understanding of the molecular defects of these disorders have permitted a foundation for classification based on molecular biology. This review presents a historical perspective on the classification of the muscular dystrophies, and furnishes the underpinnings of a genetic classification that can be used both at the bedside and in the research laboratory.

Published
1999
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