How children and caregivers viewed the change from nusinersen to risdiplam for treating spinal muscular atrophy

被引：4

作者：

Agosto, Caterina ^{[1
]}

Benedetti, Francesca ^{[2
]}

Salamon, Eleonora ^{[1
]}

Mercante, Anna ^{[3
]}

Papa, Simonetta ^{[4
]}

Giacomelli, Luca ^{[4
]}

Santini, Anna ^{[1
]}

Benini, Franca ^{[1
]}

机构：

[1] Padua Univ Hosp, Dept Womens & Childrens Hlth, Pediat Pain & Palliat Care Serv, Padua, Italy

[2] Univ Padua, Pediat Residency Program, Padua, Italy

[3] Univ Padua, Child Neuropsychiat Residency Program, Padua, Italy

[4] Polistudium SRL, Milan, Italy

来源：

ACTA PAEDIATRICA | 2023年 / 112卷 / 02期

关键词：

D O I：

10.1111/apa.16568

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

[No abstract available]

引用

页码：311 / 312

页数：2

共 5 条

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Shah, Aashni ;

Perilongo, Giorgio ;

Benini, Franca .

ORPHANET JOURNAL OF RARE DISEASES, 2021, 16 (01)

[2] Age and baseline values predict 12 and 24-month functional changes in type 2 SMA [J].

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Pera, Maria C. ;

Lucibello, Simona ;

Montes, Jacqueline ;

Pasternak, Amy ;

Mayhew, Anna ;

Glanzman, Allan M. ;

Young, Sally Dunaway ;

Pane, Marika ;

Scoto, Mariacristina ;

Messina, Sonia ;

Goemans, Nathalie ;

Osorio, Andres Nascimiento ;

Pedemonte, Marina ;

Sansone, Valeria ;

Bertini, Enrico ;

De Vivo, Darryl C. ;

Finkel, Richard ;

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[3]

Li Q, 2020, YONSEI MED J, V61, P273, DOI [10.3349/ymj.2020.61.4273, 10.3349/ymj.2020.61.4.273]

[4] Risdiplam: an investigational survival motor neuron 2 (SMN2) splicing modifier for spinal muscular atrophy (SMA) [J].

Markati, Theodora ;

Fisher, Gemma ;

Ramdas, Sithara ;

Servais, Laurent .

EXPERT OPINION ON INVESTIGATIONAL DRUGS, 2022, 31 (05) :451-461

[5]

Streiner D, 2015, HLTH MEASUREMENT SCA

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