O.16 Current progress and preliminary results with the systemic administration trial of AVI-4658, a novel phosphorodiamidate morpholino oligomer (PMO) skipping dystrophin exon 51 in Duchenne muscular dystrophy (DMD)

S.B. Shrewsbury; S. Cirak; M. Guglieri; K. Bushby; F. Muntoni

doi:10.1016/j.nmd.2010.07.140

O.16 Current progress and preliminary results with the systemic administration trial of AVI-4658, a novel phosphorodiamidate morpholino oligomer (PMO) skipping dystrophin exon 51 in Duchenne muscular dystrophy (DMD)

Neuromuscular Disorders ◽

10.1016/j.nmd.2010.07.140 ◽

2010 ◽

Vol 20 (9-10) ◽

pp. 639-640 ◽

Cited By ~ 3

Author(s):

S.B. Shrewsbury ◽

S. Cirak ◽

M. Guglieri ◽

K. Bushby ◽

F. Muntoni

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Systemic Administration ◽

Preliminary Results

Download Full-text

Duchenne muscular dystrophy: preliminary results of the Risbo-DMD study

Bone Abstracts ◽

10.1530/boneabs.7.p28 ◽

2019 ◽

Author(s):

Francesca Broggi ◽

Silvia Vai ◽

Giovanni Baranello ◽

Valeria Sansone Sansone ◽

Grazia D'Angelo ◽

...

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Preliminary Results

Download Full-text

Faculty Opinions recommendation of Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy.

Faculty Opinions – Post-Publication Peer Review of the Biomedical Literature ◽

10.3410/f.733068497.793560204 ◽

2019 ◽

Author(s):

Sue Fletcher

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Antisense Oligonucleotide ◽

Systemic Administration

Download Full-text

P4.9 A phase 2, randomized, placebo-controlled, multiple ascending-dose study of ACE-031 (ActRIIB-IgG1) in Duchenne muscular dystrophy (DMD): Preliminary results

Neuromuscular Disorders ◽

10.1016/j.nmd.2011.06.974 ◽

2011 ◽

Vol 21 (9-10) ◽

pp. 707

Author(s):

C. Campbell ◽

K.M. Attie ◽

J.K. Mah ◽

M. Tarnopolsky ◽

K. Selby ◽

...

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Phase 2 ◽

Preliminary Results ◽

Dose Study

Download Full-text

MON-LB050 Preliminary Results from the "Observational Study of Clinical Outcomes for Testosterone Treatment of Pubertal Delay in Duchenne Muscular Dystrophy (DMD) (NCT 02571205)"

Journal of the Endocrine Society ◽

10.1210/js.2019-mon-lb050 ◽

2019 ◽

Vol 3 (Supplement_1) ◽

Author(s):

Claire Wood ◽

Volker Straub ◽

Michela Guglieri ◽

Tim Cheetham

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Observational Study ◽

Clinical Outcomes ◽

Testosterone Treatment ◽

Preliminary Results ◽

Pubertal Delay

Download Full-text

Large scale population screening for Duchenne muscular dystrophy – Preliminary results

American Journal of Obstetrics and Gynecology ◽

10.1016/j.ajog.2021.11.583 ◽

2022 ◽

Vol 226 (1) ◽

pp. S347

Author(s):

Gal Cohen ◽

Atalia Shtorch-Asor ◽

Racheli Goldfarb-Yaacobi ◽

Meirav Kaiser ◽

Revital Rosenfeld ◽

...

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Large Scale ◽

Population Screening ◽

Preliminary Results ◽

Scale Population

Download Full-text

S.P.63 Using a novel ambulatory Ankle Foot Orthoses and Footwear Combination (NAFOFC) in Duchenne Muscular Dystrophy (DMD): Preliminary results for acceptability and changes in functional participation

Neuromuscular Disorders ◽

10.1016/j.nmd.2012.06.281 ◽

2012 ◽

Vol 22 (9-10) ◽

pp. 888

Author(s):

W. Bromwich ◽

M. James ◽

C. Stewart ◽

N. Emery ◽

R. Quinlivan

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Foot Orthoses ◽

Preliminary Results ◽

Ankle Foot Orthoses

Download Full-text

Exon 53 skipping of the dystrophin gene in patients with Duchenne muscular dystrophy by systemic administration of NS-065/NCNP-01: A phase 1, dose escalation, first-in-human study

Neuromuscular Disorders ◽

10.1016/j.nmd.2015.06.276 ◽

2015 ◽

Vol 25 ◽

pp. S261-S262 ◽

Cited By ~ 3

Author(s):

H. Komaki ◽

T. Nagata ◽

T. Saito ◽

S. Masuda ◽

E. Takeshita ◽

...

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Dose Escalation ◽

Phase 1 ◽

Human Study ◽

Dystrophin Gene ◽

Systemic Administration

Download Full-text

P01 Current progress with the systemic administration trial of AVI-4658, a novel Phosphorodiamidate Morpholino Oligomer (PMO) skipping dystrophin exon 51 in Duchenne muscular dystrophy (DMD)

Neuromuscular Disorders ◽

10.1016/s0960-8966(10)70016-x ◽

2010 ◽

Vol 20 ◽

pp. S5

Author(s):

S. Cirak ◽

M. Guglieri ◽

S. Shrewsbury ◽

K. Bushby ◽

F. Muntoni

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Systemic Administration

Download Full-text

Impact of Bisphosphonates on Survival for Patients With Duchenne Muscular Dystrophy

Yearbook of Neurology and Neurosurgery ◽

10.1016/j.yneu.2011.04.066 ◽

2011 ◽

Vol 2011 ◽

pp. 198-199

Author(s):

G.N. Breningstall

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy

Download Full-text

Treatment of Duchenne muscular dystrophy with cyclosporin A – A randomized, double-blind, placebo controlled trial

Neuropediatrics ◽

10.1055/s-0029-1215757 ◽

2008 ◽

Vol 39 (05) ◽

Author(s):

J Kirschner ◽

J Schessl ◽

G Ihorst ◽

R Korinthenberg

Keyword(s):

Duchenne Muscular Dystrophy ◽

Muscular Dystrophy ◽

Cyclosporin A ◽

Controlled Trial ◽

Double Blind ◽

Double Blind Placebo

Download Full-text