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Spinal Muscular Atrophy Type 1, Werdnig-Hoffmann Disease
Genetic Neuromuscular Disorders
◽
10.1007/978-3-319-56454-8_86
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2017
◽
pp. 341-343
Author(s):
Corrado Angelini
Keyword(s):
Spinal Muscular Atrophy
◽
Muscular Atrophy
◽
Spinal Muscular Atrophy Type
◽
Werdnig Hoffmann
Download Full-text
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References
Spinal Muscular Atrophy Type 1, Werdnig-Hoffmann Disease
Genetic Neuromuscular Disorders
◽
10.1007/978-3-319-07500-6_66
◽
2014
◽
pp. 295-297
Author(s):
Corrado Angelini
Keyword(s):
Spinal Muscular Atrophy
◽
Muscular Atrophy
◽
Spinal Muscular Atrophy Type
◽
Werdnig Hoffmann
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Migrating atelectasis in Werdnig-Hoffmann disease: Pulmonary manifestations in two cases of spinal muscular atrophy type 1
Pediatric Pulmonology
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10.1002/(sici)1099-0496(199908)28:2<149::aid-ppul12>3.0.co;2-w
◽
1999
◽
Vol 28
(2)
◽
pp. 149-153
◽
Cited By ~ 7
Author(s):
E.A. Leistikow
◽
N.E. Jones
◽
K.D. Josephson
◽
T.M. de Sierra
◽
D.T. Costakos
◽
...
Keyword(s):
Spinal Muscular Atrophy
◽
Muscular Atrophy
◽
Spinal Muscular Atrophy Type
◽
Pulmonary Manifestations
◽
Werdnig Hoffmann
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Adult-onset motor neuron disease and infantile Werdnig-Hoffmann disease (spinal muscular atrophy type 1) in the same family
Neurology
◽
10.1212/wnl.42.8.1477
◽
1992
◽
Vol 42
(8)
◽
pp. 1477-1477
◽
Cited By ~ 13
Author(s):
P. J. Shaw
◽
P. G. Ince
◽
J. Goodship
◽
J. Burn
◽
J. Slade
◽
...
Keyword(s):
Spinal Muscular Atrophy
◽
Motor Neuron
◽
Motor Neuron Disease
◽
Muscular Atrophy
◽
Adult Onset
◽
Spinal Muscular Atrophy Type
◽
Werdnig Hoffmann
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FV 953. Parents’ Experiences during the Compassionate Use Program (Nusinersen) for Patients with Spinal Muscular Atrophy Type 1—A Qualitative Interview Study
10.1055/s-0038-1675931
◽
2018
◽
Author(s):
Thorsten Langer
◽
Petra Kiefer
◽
Astrid Pechmann
◽
Janbernd Kirschner
Keyword(s):
Spinal Muscular Atrophy
◽
Muscular Atrophy
◽
Qualitative Interview
◽
Interview Study
◽
Compassionate Use
◽
Spinal Muscular Atrophy Type
◽
Qualitative Interview Study
◽
Parents Experiences
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Study Design of STR1VE-EU, a Phase 3 Trial of AVXS-101 Gene-Replacement Therapy (GRT) in Patients With Spinal Muscular Atrophy Type 1 (SMA1) in Europe
10.1055/s-0039-1698171
◽
2019
◽
Author(s):
Francesco Muntoni
◽
Giovanni Baranello
◽
Claudio Bruno
◽
Stefania Corti
◽
Riccardo Masson
◽
...
Keyword(s):
Spinal Muscular Atrophy
◽
Replacement Therapy
◽
Study Design
◽
Muscular Atrophy
◽
Gene Replacement
◽
Phase 3
◽
Spinal Muscular Atrophy Type
◽
Gene Replacement Therapy
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Survival of Patients With Spinal Muscular Atrophy Type 1
PEDIATRICS
◽
10.1542/peds.2012-2278d
◽
2013
◽
Vol 131
(5)
◽
pp. X31-X31
Keyword(s):
Spinal Muscular Atrophy
◽
Muscular Atrophy
◽
Spinal Muscular Atrophy Type
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Long‐term benefits of nusinersen in a child affected by cystic fibrosis and spinal muscular atrophy type 1
Pediatric Pulmonology
◽
10.1002/ppul.25334
◽
2021
◽
Author(s):
Giorgia Bruno
◽
Bernadette Donnarumma
◽
Alessia Inverardi
◽
Paolo Buonpensiero
◽
Angela Sepe
◽
...
Keyword(s):
Cystic Fibrosis
◽
Spinal Muscular Atrophy
◽
Muscular Atrophy
◽
Spinal Muscular Atrophy Type
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Brain, cognition, and language development in spinal muscular atrophy type 1: a scoping review
Developmental Medicine & Child Neurology
◽
10.1111/dmcn.14798
◽
2021
◽
Author(s):
Riccardo Masson
◽
Chiara Brusa
◽
Mariacristina Scoto
◽
Giovanni Baranello
Keyword(s):
Language Development
◽
Spinal Muscular Atrophy
◽
Scoping Review
◽
Muscular Atrophy
◽
Spinal Muscular Atrophy Type
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Nusinersen in spinal muscular atrophy type 1 from neonates to young adult: 1-year data from three Asia-Pacific regions
Journal of Neurology Neurosurgery & Psychiatry
◽
10.1136/jnnp-2020-324532
◽
2021
◽
pp. jnnp-2020-324532
Author(s):
Sophelia Hoi-Shan Chan
◽
Jong-Hee Chae
◽
Yin-Hsiu Chien
◽
Tae-Sung Ko
◽
Jee Hun Lee
◽
...
Keyword(s):
Young Adult
◽
Spinal Muscular Atrophy
◽
Muscular Atrophy
◽
Asia Pacific
◽
Spinal Muscular Atrophy Type
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Choosing Life with Spinal Muscular Atrophy Type 1
Advances in Therapy
◽
10.1007/s12325-020-01340-y
◽
2020
◽
Vol 37
(5)
◽
pp. 1708-1713
Author(s):
Moran Lavie
◽
Hodaya Nisnkorn
◽
Liora Sagi
◽
Israel Amirav
Keyword(s):
Spinal Muscular Atrophy
◽
Muscular Atrophy
◽
Spinal Muscular Atrophy Type
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