U.S. Patient Advocacy Groups
(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…
NEUROLOGY
Pharmaceutical Products and Non-pharmaceutical Interventions as Potential Treatments for Patients with Muscular Dystrophy
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…
Key Challenges to the Approval of Products to Treat Patients with Muscular Dystrophy
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:…
Global Regulatory Landscape
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…
Orthopaedic Management of the Child with Muscular Dystrophy
© 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…
An Overview of the Other Muscular Dystrophies: Underlying Genetic and Molecular Mechanisms
© 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,…
Physical Therapy and Orthotic Devices
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)…
Overview of Current Treatments for Muscular Dystrophy
© 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…
FSHD: The Most Common Type of Muscular Dystrophy?
© 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,…
Muscular Dystrophy: Historical Background and Types
© 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…
