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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G140(P) Ethical considerations and challenges in long term ventilation decisions in children with spinal muscular atrophy Type –1

Archives of Disease in Childhood ◽

10.1136/archdischild-2016-310863.131 ◽

2016 ◽

Vol 101 (Suppl 1) ◽

pp. A74.1-A74

Author(s):

C Kanaris

Keyword(s):

Spinal Muscular Atrophy ◽

Muscular Atrophy ◽

Ethical Considerations ◽

Spinal Muscular Atrophy Type ◽

Long Term Ventilation

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P.351Gene-replacement therapy (GRT) in spinal muscular atrophy type 1 (SMA1): long-term follow-up from the onasemnogene abeparvovec phase 1/2a clinical trial

Neuromuscular Disorders ◽

10.1016/j.nmd.2019.06.513 ◽

2019 ◽

Vol 29 ◽

pp. S184

Author(s):

J. Mendell ◽

R. Shell ◽

K. Lehman ◽

M. McColly ◽

L. Lowes ◽

...

Keyword(s):

Clinical Trial ◽

Spinal Muscular Atrophy ◽

Replacement Therapy ◽

Muscular Atrophy ◽

Phase 1 ◽

Spinal Muscular Atrophy Type ◽

Long Term Follow Up

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Lethal Cardiac Complications in a Long-Term Survivor of Spinal Muscular Atrophy Type 1

Kosin Medical Journal ◽

10.7180/kmj.2019.34.1.47 ◽

2019 ◽

Vol 34 (1) ◽

pp. 47

Author(s):

Min-Jung Cho

Keyword(s):

Spinal Muscular Atrophy ◽

Muscular Atrophy ◽

Cardiac Complications ◽

Spinal Muscular Atrophy Type ◽

Term Survivor

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POINT: Is Noninvasive Ventilation Always the Most Appropriate Manner of Long-term Ventilation for Infants With Spinal Muscular Atrophy Type 1? Yes, Almost Always

CHEST Journal ◽

10.1016/j.chest.2016.11.043 ◽

2017 ◽

Vol 151 (5) ◽

pp. 962-965 ◽

Cited By ~ 16

Author(s):

John R. Bach

Keyword(s):

Spinal Muscular Atrophy ◽

Noninvasive Ventilation ◽

Muscular Atrophy ◽

Spinal Muscular Atrophy Type ◽

Long Term Ventilation

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S11 Long-term follow-up of the phase 1 START trial of onasemnogene abeparvovec gene therapy in spinal muscular atrophy type 1

10.1136/thorax-2020-btsabstracts.17 ◽

2021 ◽

Cited By ~ 1

Author(s):

JR Mendell ◽

R Shell ◽

KJ Lehman ◽

M McColly ◽

LP Lowes ◽

...

Keyword(s):

Gene Therapy ◽

Spinal Muscular Atrophy ◽

Muscular Atrophy ◽

Phase 1 ◽

Spinal Muscular Atrophy Type ◽

Long Term Follow Up ◽

Start Trial

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COUNTERPOINT: Is Noninvasive Ventilation Always the Most Appropriate Manner of Long-term Ventilation for Infants With Spinal Muscular Atrophy Type 1? No

CHEST Journal ◽

10.1016/j.chest.2016.11.039 ◽

2017 ◽

Vol 151 (5) ◽

pp. 965-968 ◽

Cited By ~ 3

Author(s):

Howard B. Panitch

Keyword(s):

Spinal Muscular Atrophy ◽

Noninvasive Ventilation ◽

Muscular Atrophy ◽

Spinal Muscular Atrophy Type ◽

Long Term Ventilation

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Spinal muscular atrophy type 1: Is long-term mechanical ventilation ethical?

Journal of Paediatrics and Child Health ◽

10.1111/j.1440-1754.2007.01052.x ◽

2007 ◽

Vol 43 (4) ◽

pp. 237-242 ◽

Cited By ~ 20

Author(s):

Monique M Ryan ◽

Henry Kilham ◽

Stephen Jacobe ◽

Bernadette Tobin ◽

David Isaacs

Keyword(s):

Mechanical Ventilation ◽

Spinal Muscular Atrophy ◽

Muscular Atrophy ◽

Spinal Muscular Atrophy Type

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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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Gene-Replacement Therapy (GRT) in Spinal Muscular Atrophy Type 1 (SMA1): Long-Term Follow-Up From the Onasemnogene Abeparvovec Phase 1/2 Clinical Trial

10.1055/s-0039-1698259 ◽

2019 ◽

Cited By ~ 1

Author(s):

Jerry R. Mendell ◽

Richard Shell ◽

Kelly J. Lehman ◽

Markus McColly ◽

Linda P. Lowes ◽

...

Keyword(s):

Clinical Trial ◽

Muscular Atrophy ◽

Phase 1 ◽

Gene Replacement ◽

Spinal Muscular Atrophy Type ◽

Gene Replacement Therapy ◽

Long Term Follow Up

Download Full-text