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1 . Academic Journal
Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial
저자
by
Acsadi, Gyula
;
Baranello, Giovanni
;
Blaschek, Astrid
;
Brandsema, John
;
Brogna, Claudia
, et al.
소스
In
The Lancet Neurology
April 2024 23(4):393-403
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7 . Academic Journal
Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial
저자
by
Volpe, Joseph J.
;
Posner, John
;
Kellner, Ulrich
;
Quinlivan, Rosaline
;
Daron, Aurore
, et al.
소스
In
The Lancet Neurology
January 2022 21(1):42-52
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7 . Academic Journal
The nonsense mutation stop+4 model correlates with motor changes in Duchenne muscular dystrophy
저자
by
Brogna, Claudia
;
Coratti, Giorgia
;
Rossi, Rachele
;
Neri, Marcella
;
Messina, Sonia
, et al.
소스
In
Neuromuscular Disorders
June 2021 31(6):479-488
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7 . Academic Journal
Six‐minute walk test as outcome measure of fatigability in adults with spinal muscular atrophy treated with nusinersen.
저자
by
Govoni, Alessandra
;
Ricci, Giulia
;
Bonanno, Silvia
;
Bello, Luca
;
Magri, Francesca
, et al.
소스
Muscle & Nerve
. Aug2024, p1. 8p. 2 Illustrations.
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7 . Academic Journal
Diffusion tensor imaging reveals subclinical alterations in muscles of patients with Becker muscular dystrophy.
저자
by
Nava, Simone
;
Conte, Giorgio
;
Triulzi, Fabio M
;
Comi, Giacomo P
;
Magri, Francesca
, et al.
소스
British Journal of Radiology
. May2024, Vol. 97 Issue 1157, p947-953. 7p.
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7 . Academic Journal
Cognitive abnormalities in Becker muscular dystrophy: a mysterious link between dystrophin deficiency and executive functions.
저자
by
Pezzoni, Laura
;
Brusa, Roberta
;
Difonzo, Teresa
;
Magri, Francesca
;
Velardo, Daniele
, et al.
소스
Neurological Sciences
. Apr2024, Vol. 45 Issue 4, p1691-1698. 8p.
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7 . Academic Journal
Pathogenic TNNI1 variants disrupt sarcomere contractility resulting in hypo- and hypercontractile muscle disease.
저자
by
Donkervoort, Sandra
;
van de Locht, Martijn
;
Ronchi, Dario
;
Reunert, Janine
;
McLean, Catriona A.
, et al.
소스
Science Translational Medicine; 4/3/2024, Vol. 16 Issue 741, p1-13, 13p
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7 . Academic Journal
Histological effects of givinostat in boys with Duchenne muscular dystrophy
저자
by
Bettica, Paolo
;
Petrini, Stefania
;
D'Oria, Valentina
;
D'Amico, Adele
;
Catteruccia, Michela
, et al.
소스
In
Neuromuscular Disorders
October 2016 26(10):643-649
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7 . Academic Journal
Therapeutic Development in Amyotrophic Lateral Sclerosis
저자
by
Bucchia, Monica
;
Ramirez, Agnese
;
Parente, Valeria
;
Simone, Chiara
;
Nizzardo, Monica
, et al.
소스
In
Clinical Therapeutics
1 March 2015 37(3):668-680
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7 . Academic Journal
Case report: A novel ACTA1 variant in a patient with nemaline rods and increased glycogen deposition.
저자
by
Piga, Daniela
;
Rimoldi, Martina
;
Magri, Francesca
;
Zanotti, Simona
;
Napoli, Laura
, et al.
소스
Frontiers in Neurology; 2024, p1-9, 9p
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