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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
Ligamentous laxity in children with achondroplasia: Prevalence, joint involvement, and implications for early intervention strategies
저자
by
Romeo, Domenico Marco
;
Pironi, Virginia
;
Velli, Chiara
;
Sforza, Elisabetta
;
Rigante, Donato
, et al.
소스
In
European Journal of Medical Genetics
April 2024 68
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7 . Academic Journal
Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy
저자
by
Mendell, Jerry R.
;
Proud, Crystal
;
Zaidman, Craig M.
;
Mason, Stefanie
;
Darton, Eddie
, et al.
소스
In
Pediatric Neurology
April 2024 153:11-18
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7 . Academic Journal
Gain and loss of upper limb abilities in Duchenne muscular dystrophy patients: A 24-month study
저자
by
Coratti, Giorgia
;
Pane, Marika
;
Brogna, Claudia
;
D'Amico, Adele
;
Pegoraro, Elena
, et al.
소스
In
Neuromuscular Disorders
January 2024 34:75-82
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7 . Academic Journal
Profile of cognitive abilities in spinal muscular atrophy type II and III: what is the role of motor impairment?
저자
by
Buchignani, Bianca
;
Cicala, Gianpaolo
;
Moriconi, Federica
;
Ricci, Martina
;
Capasso, Anna
, et al.
소스
In
Neuromuscular Disorders
October 2023 33(10):711-717
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7 . Academic Journal
Psychological test usage in duchenne muscular dystrophy: An EU multi-centre study
저자
by
Weerkamp, Pien
;
Chieffo, Daniela
;
Collin, Philippe
;
Moriconi, Federica
;
Papageorgiou, Andriani
, et al.
소스
In
European Journal of Paediatric Neurology
September 2023 46:42-47
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7 . Academic Journal
Patient reported outcome measure for upper limb in Duchenne muscular dystrophy: correlation with PUL2.0.
저자
by
Cicala, Gianpaolo
;
Pane, Marika
;
Coratti, Giorgia
;
Brogna, Claudia
;
Fanelli, Lavinia
, et al.
소스
In
Neuromuscular Disorders
September 2023 33(9):69-73
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7 . Academic Journal
Safety, tolerability and pharmacokinetics of eteplirsen in young boys aged 6–48 months with Duchenne muscular dystrophy amenable to exon 51 skipping
저자
by
Deconinck, Nicolas
;
Van Coster, Rudy
;
Vanlander, Arnaud
;
Seferian, Andreea
;
De Lucia, Silvana
, et al.
소스
In
Neuromuscular Disorders
June 2023 33(6):476-483
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7 . Academic Journal
Onasemnogene abeparvovec in spinal muscular atrophy: predictors of efficacy and safety in naïve patients with spinal muscular atrophy and following switch from other therapies
저자
by
Pera, Maria Carmela
;
Bravetti, Chiara
;
Piastra, Marco
;
Genovese, Orazio
;
Cicala, Gianpaolo
, et al.
소스
In
eClinicalMedicine
May 2023 59
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7 . Academic Journal
Long term follow-up of scoliosis progression in type II SMA patients
저자
by
Coratti, Giorgia
;
Pera, Maria Carmela
;
D'Amico, Adele
;
Bruno, Claudio
;
Bovis, Francesca
, et al.
소스
In
Neuromuscular Disorders
December 2022 32(11-12):879-885
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