European ad-hoc consensus statement on gene replacement therapy for spinal muscular atrophy

Authors:

Jodi M. Wolffa ∙ Nora Capoccib ∙ Evrim Atasb ∙ Diana X. Bharucha-Goebelc ∙ John F. Brandsemad ∙ Russell J. Butterfielde ∙ Christina B. Chadwickf ∙ Manuela Cortif,g ∙ Thomas O. Crawfordh ∙ Linda Cripei ∙ John W. Dayj ∙ Tina Duongj ∙ Mai K. ElMallahk ∙ Kevin M. Flanigani,l ∙ Lindsey A. Georgem ∙ Natalie L. Goedekern ∙ Erica Goudeo ∙ Sharon Hesterleeb ∙ Brian Linb ∙ Natalie K. Katzk ∙ Susan E. Matesanzd ∙ Craig McDonaldo ∙ Elizabeth M. McNallyp ∙ Claudia Mercado-Rodriguezf,g ∙ Deipanjan Nandiq ∙ Julie A. Parsonsr ∙ Crystal Prouds ∙ Leigh Ramos-Plattt ∙ Angela Lekb ∙ Stephanie M. Salabarriaf,g ∙ Eric Caminoa ∙ Rachel Schradera ∙ Erin Sheak ∙ Richard Shelli ∙ Perry B. Shiehu ∙ Jonathan H. Soslowv ∙ Jane B. Taylorw ∙ Aravindhan Veerapandiyanx ∙ Chet Villay ∙ Michele L. Yangx ∙ Craig M. Zaidmann ∙ Carmen Leon-Astudillof,g ∙ Barry J. Byrnef,g Barry.byrne@ufl.edu Show less
Affiliations & Notes
a
Parent Project Muscular Dystrophy, USA
b
Muscular Dystrophy Association, USA
c
Neuromuscular and Neurogenetic Disorders of Childhood, NINDS/NIH, Bethesda, MD, USA, Division of Neurology, Children’s National Hospital, WA, DC, USA
d
Division of Neurology, Children’s Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, PA, USA
e
Department of Pediatric Neurology, University of Utah, Salt Lake City, UT, USA
f
Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA
g
Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, FL, USA
h
Department of Pediatrics, Johns Hopkins University, Baltimore, MD, USA
i
Department of Pediatrics and Neurology, Nationwide Children’s Hopsital, Columbus, OH, USA
j
Department of Neurology, Stanford University, Stanford, CA, USA
k
Department of Pediatrics, School of Medicine, Duke University, Durham, NC, USA
l
Center for Gene Therapy, Nationwide Children’s Hospital, Columbus, OH, USA
m
Department of Pediatrics, Children’s Hospital of Philadelphia, Perelman School of Medicine, University of Pennsylvania, Philadelphia PA, USA
n
Department of Neurology, Washington University in St. Louis School of Medicine, St. Louis, MO, USA
o
Department of Physical Medicine and Rehabilitation, University of California Davis School of Medicine, Sacramento, CA, USA
p
Center for Genetic Medicine, Bluhm Cardiovascular Institute, Northwestern University Feinberg School of Medicine, Chicago, IL, USA
q
The Heart Center, Nationwide Children’s Hospital, Columbus, OH, USA
r
Children’s Hospital Colorado, University of Colorado School of Medicine, Aurora, CO, USA
s
Children’s Hospital of the King’s Daughters, Norfolk, VA, USA
t
Department of Pediatrics, Keck School of Medicine, University of Southern California and Children’s Hospital of Los Angeles, Los Angeles, CA, USA
u
Department of Pediatrics, University of California, Los Angeles, CA USA
v
Division of Pediatric Cardiology, Vanderbilt University Medical Center, Nashville, TN, USA
w
Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA
x
Department of Pediatrics, University of Arkansas for Medical Sciences, Arkansas Children’s Hospital, Little Rock, AR, USA
y
The Heart Institute, Department of Pediatrics, Cincinnati Children’s Hospital Medical Center, Cincinnati, OH, USA