© Springer International Publishing Switzerland 2015Raymond A. Huml (ed.)Muscular Dystrophy10.1007/978-3-319-17362-7_15 15. Summary Raymond A. Huml1 (1) Biosimilars Center of Excellence, Quintiles Inc., 4820 Emperor Boulevard, Durham, NC 27703, USA Raymond A. Huml Email: raymond.huml@quintiles.com Muscular dystrophy (MD)…
© Springer International Publishing Switzerland 2015Raymond A. Huml (ed.)Muscular Dystrophy10.1007/978-3-319-17362-7_14 14. Global and National Patient Registries Raymond A. Huml1 (1) Biosimilars Center of Excellence, Quintiles Inc., 4820 Emperor Boulevard, Durham, NC 27703, USA Raymond A. Huml Email:…
(1) ℅ Raymond A. Huml, Quintiles Inc., 4820 Emperor Blvd, Durham, NC 27703, USA For newly diagnosed muscular dystrophy (MD) patients and their caregivers in the U.S., the first point…
Fig. 12.1 FDA foundation building for clinical trial drug development As the scientific (e.g., pathophysiology, mechanism of action [MOA], effect of intervention) and clinical data (post IND phase, such as…
Name of document Date released Report: complex issues in developing drugs and biological products for rare diseases and accelerating the development of therapies for pediatric rare diseases including strategic plan:…
Document Reference number Publication date Concept paper on the need for a guideline on the treatment of Duchenne and Becker muscular dystrophy EMA/CHMP/CNSWP/236981/2011 – Adoption by CHMP for release for…
© Springer International Publishing Switzerland 2015Raymond A. Huml (ed.)Muscular Dystrophy10.1007/978-3-319-17362-7_9 9. Orthopaedic Management of the Child with Muscular Dystrophy Robert K. Lark1 and Elizabeth W. Hubbard1 (1) Department of Orthopaedic Surgery, Duke University Medical Center, Lenox Baker Children’s…
© Springer International Publishing Switzerland 2015Raymond A. Huml (ed.)Muscular Dystrophy10.1007/978-3-319-17362-7_5 5. An Overview of the Other Muscular Dystrophies: Underlying Genetic and Molecular Mechanisms Jean K. Mah1 (1) Department of Pediatrics and Clinical Neurosciences, University of Calgary,…
DMD/BMD [2, 3, 6, 19, 30, 31, 33, 44] DMD/BMD weakness, as detailed in Chapter 4, tends to be symmetrical Characteristic weakness (early stage) Characteristic compensations/posture/patterns of movement (early stage)…
© Springer International Publishing Switzerland 2015Raymond A. Huml (ed.)Muscular Dystrophy10.1007/978-3-319-17362-7_7 7. Overview of Current Treatments for Muscular Dystrophy Zheng (Jane) Fan1 (1) Department of Neurology, University of North Carolina at Chapel Hill, CB# 7025, 2145 Physicians…
