Response to “Railroading at the FDA”

Approving a Problematic Muscular Dystrophy Drug

(2016) Aaron S. Kesselheim et al.   JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION

Railroading at the FDA

(2016)   NATURE BIOTECHNOLOGY

Railroading at the FDA

(2016)   NATURE MEDICINE

DMDgenotypes and loss of ambulation in the CINRG Duchenne Natural History Study

(2016) Luca Bello et al.   NEUROLOGY

Dystrophin quantification: Biological and translational research implications

(2014) K. Anthony et al.   NEUROLOGY

Eteplirsen for the treatment of Duchenne muscular dystrophy

(2013) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Low dystrophin levels increase survival and improve muscle pathology and function in dystrophin/utrophin double-knockout mice

(2013) Maaike van Putten et al.   FASEB JOURNAL

A marginal level of dystrophin partially ameliorates hindlimb muscle passive mechanical properties in dystrophin-null mice

(2012) Chady H. Hakim et al.   MUSCLE & NERVE

Quantification of dystrophin immunofluorescence in dystrophinopathy muscle specimens

(2012) L. E. Taylor et al.   NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY

The Effects of Low Levels of Dystrophin on Mouse Muscle Function and Pathology

(2012) Maaike van Putten et al.   PLoS One

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study

(2009) Maria Kinali et al.   LANCET NEUROLOGY

Immunohistological intensity measurements as a tool to assess sarcolemma-associated protein expression

(2009) V. Arechavala-Gomeza et al.   NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY

Preservation of Muscle Force in Mdx3cv Mice Correlates with Low-Level Expression of a Near Full-Length Dystrophin Protein

(2008) Dejia Li et al.   AMERICAN JOURNAL OF PATHOLOGY