Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

A Randomized Placebo-Controlled Phase 3 Trial of an Antisense Oligonucleotide, Drisapersen, in Duchenne Muscular Dystrophy

Goemans, Nathalie
Mercuri, Eugenio
Belousova, Elena
Komaki, Hirofumi
Dubrovsky, Alberto
McDonald, Craig M
Kraus, John E
Lourbakos, Afrodite
Lin, Zhengning
Campion, Giles
Wang, Susanne X
Campbell, Craig
DEMAND III study group

January 2018

This 48-week, randomized, placebo-controlled phase 3 study (DMD114044; NCT01254019) evaluated effica...

Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study.

Nathalie M Goemans
Már Tulinius
Marleen van den Hauwe
Anna-Karin Kroksmark
Gunnar Buyse
Rosamund J Wilson
Judith C van Deutekom
Sjef J de Kimpe
Afrodite Lourbakos
Giles Campion

January 2016

BackgroundDrisapersen induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthe...

G.P.147 - Outcome measures for Duchenne muscular dystrophy from ambulant to non-ambulant: implications for clinical trials

Ricotti, V
Eagle, M
Butler, J
Decostre, V
Deborah, R
Moraux, A
Anthony, Karen
Sleby, V
Guglieri, M
Van der Holst, M
Jansen, M
Morgan, J
de Groot, I
Niks, E
Verschuuren, J
Servais, L
Hogrel, J Y
Voit, T
Straub, V
Muntoni, F

October 2015

Novel emerging therapies for Duchenne muscular dystrophy (DMD), such as antisense oligomer (AO) medi...

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

Mendell, Jerry R
Goemans, Nathalie
Lowes, Linda P
Alfano, Lindsay N
Berry, Katherine
Shao, James
Kaye, Edward M
Mercuri Eugenio
Battini Roberta

January 2016

To continue evaluation of the long-term efficacy and safety of eteplirsen, a phosphorodiamidate morp...

Eteplirsen in the treatment of Duchenne muscular dystrophy

Lim KRQ
Maruyama R
Yokota T

February 2017

Kenji Rowel Q Lim,1 Rika Maruyama,1 Toshifumi Yokota1,2 1Department of Medical Genetics, Faculty of...

Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial.

Shieh, Perry
Abdel-Hamid, Hoda
Connolly, Anne
Ciafaloni, Emma
Wagner, Kathryn
Goemans, Nathalie
Mercuri, Eugenio
Khan, Navid
Koenig, Erica
Malhotra, Jyoti
Zhang, Wenfei
Han, Baoguang
Mendell, Jerry
McDonald, Craig

January 2021

BackgroundEteplirsen received accelerated FDA approval for treatment of Duchenne muscular dystrophy ...

Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.

Servais, Laurent
Mercuri, Eugenio
Straub, Volker
Guglieri, Michela
Seferian, Andreea M.
Scoto, Mariacristina
Leone, Daniela
Koenig, Erica
Khan, Navid
Dugar, Ashish
Wang, Xiaodong
Han, Baoguang
Wang, Dan
Muntoni, Francesco

February 2022

peer reviewedThe aim of this Phase 1/2, 2-part, multicenter trial was to report clinical safety and ...

A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment.

Mitelman, Olga
Abdel-Hamid, Hoda
Byrne, Barry
Connolly, Anne
Heydemann, Peter
Proud, Crystal
Shieh, Perry
Wagner, Kathryn
Dugar, Ashish
Santra, Sourav
Signorovitch, James
Goemans, Nathalie
Mercuri, Eugenio
Mendell, Jerry
McDonald, Craig

January 2022

BACKGROUND: Studies 4658-201/202 (201/202) evaluated treatment effects of eteplirsen over 4 years in...

Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial

Servais, L
Mercuri, E
Straub, V
Guglieri, M
Seferian, AM
Scoto, M
Leone, D
Koenig, E
Khan, N
Dugar, A
Wang, X
Han, B
Wang, D
Muntoni, F
SKIP-NMD Study Group, .

November 2021

The aim of this Phase 1/2, 2-part, multicenter trial was to report clinical safety and efficacy of l...

Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison of Eteplirsen-Treated Patients to Natural History

Kinane, T. Bernard
Mayer, Oscar H.
Duda, Petra W.
Lowes, Linda P.
Moody, Stephanie L.
Mendell, Jerry R.

April 2018

Background: Duchenne muscular dystrophy (DMD) is a rare, degenerative, X-linked genetic disease that...

Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.

Clemens, Paula
Rao, Vamshi
Connolly, Anne
Harper, Amy
Mah, Jean
Smith, Edward
Zaidman, Craig
Nakagawa, Tomoyuki
Hoffman, Eric
McDonald, Craig

January 2022

BACKGROUND: Duchenne muscular dystrophy (DMD) is a rare, genetic disease caused by mutations in the ...

Comparison of ambulatory capacity and disease progression of Duchenne muscular dystrophy subjects enrolled in the drisapersen DMD114673 study with a matched natural history cohort of subjects on daily corticosteroids

Goemans, Nathalie
Tulinius, Mar
Kroksmark, Anna-Karin
Wilson, Rosamund
van den Hauwe, Marleen
Campion, Giles

January 2017

Duchenne muscular dystrophy is a rare genetic disorder with life-limiting pathology. Drisapersen ind...

Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study.

Smith, Edward
Zaidman, Craig
Nakagawa, Tomoyuki
Hoffman, Eric
Clemens, Paula
Rao, Vamshi
Connolly, Anne
Harper, Amy
Mah, Jean
McDonald, Craig

January 2023

BACKGROUND: Duchenne muscular dystrophy (DMD) is caused by DMD gene mutations, resulting in absence ...

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

Cirak, Sebahattin
Arechavala-Gomeza, Virginia
Guglieri, Michela
Feng, Lucy
Torelli, Silvia
Anthony, Karen
Abbs, Stephen
Garralda, Maria Elena
Bourke, John
Wells, Dominic J
Dickson, George
Wood, Matthew JA
Wilton, Steve D
Straub, Volker
Kole, Ryszard
Shrewsbury, Stephen B
Sewry, Caroline
Morgan, Jennifer E
Bushby, Kate
Muntoni, Francesco

August 2011

SummaryBackgroundWe report clinical safety and biochemical efficacy from a dose-ranging study of int...

Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53

Brogna, Claudia
Coratti, Giorgia
Pane, Marika
Ricotti, Valeria
Messina, Sonia
D'Amico, Adele
Bruno, Claudio
Vita, Gianluca
Berardinelli, Angela
Mazzone, Elena
Magri, Francesca
Ricci, Federica
Mongini, Tiziana
Battini, Roberta
Bello, Luca
Pegoraro, Elena
Baranello, Giovanni
Previtali, Stefano C
Politano, Luisa
Comi, Giacomo P
Sansone, Valeria A
Donati, Alice
Bertini, Enrico
Muntoni, Francesco
Goemans, Nathalie
Mercuri, Eugenio

January 2019

The aim of this international collaborative effort was to report 36-month longitudinal changes using...

A Randomized Placebo-Controlled Phase 3 Trial of an Antisense Oligonucleotide, Drisapersen, in Duchenne Muscular Dystrophy

Goemans, Nathalie
Mercuri, Eugenio
Belousova, Elena
Komaki, Hirofumi
Dubrovsky, Alberto
McDonald, Craig M
Kraus, John E
Lourbakos, Afrodite
Lin, Zhengning
Campion, Giles
Wang, Susanne X
Campbell, Craig
DEMAND III study group

January 2018

This 48-week, randomized, placebo-controlled phase 3 study (DMD114044; NCT01254019) evaluated effica...

Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study.

Nathalie M Goemans
Már Tulinius
Marleen van den Hauwe
Anna-Karin Kroksmark
Gunnar Buyse
Rosamund J Wilson
Judith C van Deutekom
Sjef J de Kimpe
Afrodite Lourbakos
Giles Campion

January 2016

BackgroundDrisapersen induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthe...

G.P.147 - Outcome measures for Duchenne muscular dystrophy from ambulant to non-ambulant: implications for clinical trials

Ricotti, V
Eagle, M
Butler, J
Decostre, V
Deborah, R
Moraux, A
Anthony, Karen
Sleby, V
Guglieri, M
Van der Holst, M
Jansen, M
Morgan, J
de Groot, I
Niks, E
Verschuuren, J
Servais, L
Hogrel, J Y
Voit, T
Straub, V
Muntoni, F

October 2015

Novel emerging therapies for Duchenne muscular dystrophy (DMD), such as antisense oligomer (AO) medi...

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

Mendell, Jerry R
Goemans, Nathalie
Lowes, Linda P
Alfano, Lindsay N
Berry, Katherine
Shao, James
Kaye, Edward M
Mercuri Eugenio
Battini Roberta

January 2016

To continue evaluation of the long-term efficacy and safety of eteplirsen, a phosphorodiamidate morp...

Eteplirsen in the treatment of Duchenne muscular dystrophy

Lim KRQ
Maruyama R
Yokota T

February 2017

Kenji Rowel Q Lim,1 Rika Maruyama,1 Toshifumi Yokota1,2 1Department of Medical Genetics, Faculty of...

Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial.

Shieh, Perry
Abdel-Hamid, Hoda
Connolly, Anne
Ciafaloni, Emma
Wagner, Kathryn
Goemans, Nathalie
Mercuri, Eugenio
Khan, Navid
Koenig, Erica
Malhotra, Jyoti
Zhang, Wenfei
Han, Baoguang
Mendell, Jerry
McDonald, Craig

January 2021

BackgroundEteplirsen received accelerated FDA approval for treatment of Duchenne muscular dystrophy ...

Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial.

Servais, Laurent
Mercuri, Eugenio
Straub, Volker
Guglieri, Michela
Seferian, Andreea M.
Scoto, Mariacristina
Leone, Daniela
Koenig, Erica
Khan, Navid
Dugar, Ashish
Wang, Xiaodong
Han, Baoguang
Wang, Dan
Muntoni, Francesco

February 2022

peer reviewedThe aim of this Phase 1/2, 2-part, multicenter trial was to report clinical safety and ...

A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment.

Mitelman, Olga
Abdel-Hamid, Hoda
Byrne, Barry
Connolly, Anne
Heydemann, Peter
Proud, Crystal
Shieh, Perry
Wagner, Kathryn
Dugar, Ashish
Santra, Sourav
Signorovitch, James
Goemans, Nathalie
Mercuri, Eugenio
Mendell, Jerry
McDonald, Craig

January 2022

BACKGROUND: Studies 4658-201/202 (201/202) evaluated treatment effects of eteplirsen over 4 years in...

Long-Term Safety and Efficacy Data of Golodirsen in Ambulatory Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A First-in-human, Multicenter, Two-Part, Open-Label, Phase 1/2 Trial

Servais, L
Mercuri, E
Straub, V
Guglieri, M
Seferian, AM
Scoto, M
Leone, D
Koenig, E
Khan, N
Dugar, A
Wang, X
Han, B
Wang, D
Muntoni, F
SKIP-NMD Study Group, .

November 2021

The aim of this Phase 1/2, 2-part, multicenter trial was to report clinical safety and efficacy of l...

Long-Term Pulmonary Function in Duchenne Muscular Dystrophy: Comparison of Eteplirsen-Treated Patients to Natural History

Kinane, T. Bernard
Mayer, Oscar H.
Duda, Petra W.
Lowes, Linda P.
Moody, Stephanie L.
Mendell, Jerry R.

April 2018

Background: Duchenne muscular dystrophy (DMD) is a rare, degenerative, X-linked genetic disease that...

Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.

Clemens, Paula
Rao, Vamshi
Connolly, Anne
Harper, Amy
Mah, Jean
Smith, Edward
Zaidman, Craig
Nakagawa, Tomoyuki
Hoffman, Eric
McDonald, Craig

January 2022

BACKGROUND: Duchenne muscular dystrophy (DMD) is a rare, genetic disease caused by mutations in the ...

Comparison of ambulatory capacity and disease progression of Duchenne muscular dystrophy subjects enrolled in the drisapersen DMD114673 study with a matched natural history cohort of subjects on daily corticosteroids

Goemans, Nathalie
Tulinius, Mar
Kroksmark, Anna-Karin
Wilson, Rosamund
van den Hauwe, Marleen
Campion, Giles

January 2017

Duchenne muscular dystrophy is a rare genetic disorder with life-limiting pathology. Drisapersen ind...

Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study.

Smith, Edward
Zaidman, Craig
Nakagawa, Tomoyuki
Hoffman, Eric
Clemens, Paula
Rao, Vamshi
Connolly, Anne
Harper, Amy
Mah, Jean
McDonald, Craig

January 2023

BACKGROUND: Duchenne muscular dystrophy (DMD) is caused by DMD gene mutations, resulting in absence ...

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

Cirak, Sebahattin
Arechavala-Gomeza, Virginia
Guglieri, Michela
Feng, Lucy
Torelli, Silvia
Anthony, Karen
Abbs, Stephen
Garralda, Maria Elena
Bourke, John
Wells, Dominic J
Dickson, George
Wood, Matthew JA
Wilton, Steve D
Straub, Volker
Kole, Ryszard
Shrewsbury, Stephen B
Sewry, Caroline
Morgan, Jennifer E
Bushby, Kate
Muntoni, Francesco

August 2011

SummaryBackgroundWe report clinical safety and biochemical efficacy from a dose-ranging study of int...

Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations amenable to skip exons 44, 45, 51 and 53

Brogna, Claudia
Coratti, Giorgia
Pane, Marika
Ricotti, Valeria
Messina, Sonia
D'Amico, Adele
Bruno, Claudio
Vita, Gianluca
Berardinelli, Angela
Mazzone, Elena
Magri, Francesca
Ricci, Federica
Mongini, Tiziana
Battini, Roberta
Bello, Luca
Pegoraro, Elena
Baranello, Giovanni
Previtali, Stefano C
Politano, Luisa
Comi, Giacomo P
Sansone, Valeria A
Donati, Alice
Bertini, Enrico
Muntoni, Francesco
Goemans, Nathalie
Mercuri, Eugenio

January 2019

The aim of this international collaborative effort was to report 36-month longitudinal changes using...

A Randomized Placebo-Controlled Phase 3 Trial of an Antisense Oligonucleotide, Drisapersen, in Duchenne Muscular Dystrophy

Goemans, Nathalie
Mercuri, Eugenio
Belousova, Elena
Komaki, Hirofumi
Dubrovsky, Alberto
McDonald, Craig M
Kraus, John E
Lourbakos, Afrodite
Lin, Zhengning
Campion, Giles
Wang, Susanne X
Campbell, Craig
DEMAND III study group

January 2018

This 48-week, randomized, placebo-controlled phase 3 study (DMD114044; NCT01254019) evaluated effica...

Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study.

Nathalie M Goemans
Már Tulinius
Marleen van den Hauwe
Anna-Karin Kroksmark
Gunnar Buyse
Rosamund J Wilson
Judith C van Deutekom
Sjef J de Kimpe
Afrodite Lourbakos
Giles Campion

January 2016

BackgroundDrisapersen induces exon 51 skipping during dystrophin pre-mRNA splicing and allows synthe...

G.P.147 - Outcome measures for Duchenne muscular dystrophy from ambulant to non-ambulant: implications for clinical trials

Ricotti, V
Eagle, M
Butler, J
Decostre, V
Deborah, R
Moraux, A
Anthony, Karen
Sleby, V
Guglieri, M
Van der Holst, M
Jansen, M
Morgan, J
de Groot, I
Niks, E
Verschuuren, J
Servais, L
Hogrel, J Y
Voit, T
Straub, V
Muntoni, F

October 2015

Novel emerging therapies for Duchenne muscular dystrophy (DMD), such as antisense oligomer (AO) medi...

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

Abstract

Extracted data

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

Abstract

Extracted data

Related items

Related items