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…
© Springer International Publishing Switzerland 2015Raymond A. Huml (ed.)Muscular Dystrophy10.1007/978-3-319-17362-7_3 3. FSHD: The Most Common Type of Muscular Dystrophy? Raymond A. Huml1 and Daniel P. Perez2 (1) Biosimilars Center of Excellence, Quintiles Inc., 4820 Emperor Boulevard, Durham, NC 27703,…
© Springer International Publishing Switzerland 2015Raymond A. Huml (ed.)Muscular Dystrophy10.1007/978-3-319-17362-7_2 2. Muscular Dystrophy: Historical Background and Types Raymond A. Huml1 (1) Biosimilars Center of Excellence, Quintiles Inc., 4820 Emperor Boulevard, Durham, NC 27703, USA Raymond A. Huml…
© Springer International Publishing Switzerland 2015Raymond A. Huml (ed.)Muscular Dystrophy10.1007/978-3-319-17362-7_6 6. Transition from Childhood to Adult in Patients with Muscular Dystrophy Elba Yesi Gerena Maldonado1 and Kathryn R. Wagner2 (1) Department of Rehabilitation Medicine, University of Washington, 1959…
