Duchenne muscular dystrophy: Drug development and regulatory considerations

2010 ◽  
Vol 41 (6) ◽  
pp. 740-745 ◽  
Author(s):  
D. Elizabeth McNeil ◽  
Carole Davis ◽  
Devanand Jillapalli ◽  
Shari Targum ◽  
Anthony Durmowicz ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Drug Development

scholarly journals Exon-Skipping in Duchenne Muscular Dystrophy

2021 ◽  
pp. 1-16
Author(s):  
Shin’ichi Takeda ◽  
Paula R. Clemens ◽  
Eric P. Hoffman
Keyword(s):  
Clinical Trials ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Drug Development ◽  
Rare Disease ◽  
19Th Century ◽  
Exon Skipping ◽  
Clinical Development ◽  
Therapeutic Development ◽  
The 19Th Century

Duchenne muscular dystrophy (DMD) is a devastating, rare disease. While clinically described in the 19th century, the genetic foundation of DMD was not discovered until more than 100 years later. This genetic understanding opened the door to the development of genetic treatments for DMD. Over the course of the last 30 years, the research that supports this development has moved into the realm of clinical trials and regulatory drug approvals. Exon skipping to therapeutically restore the frame of an out-of-frame dystrophin mutation has taken center stage in drug development for DMD. The research reviewed here focuses on the clinical development of exon skipping for the treatment of DMD. In addition to the generation of clinical treatments that are being used for patient care, this research sets the stage for future therapeutic development with a focus on increasing efficacy while providing safety and addressing the multi-systemic aspects of DMD.

scholarly journals First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Development in Neuromuscular Diseases

2021 ◽  
pp. 183-190
Author(s):  
Laurent Servais ◽  
Eric Camino ◽  
Aude Clement ◽  
Craig M. McDonald ◽  
Jacek Lukawy ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Drug Development ◽  
Neuromuscular Diseases ◽  
European Medicines Agency ◽  
Motor Abilities ◽  
Stakeholder Perspective ◽  
Functional Outcome Measures ◽  
Multi Stakeholder ◽  
The Impact

<b><i>Background:</i></b> Functional outcome measures used to assess efficacy in clinical trials of investigational treatments for rare neuromuscular diseases like Duchenne muscular dystrophy (DMD) are performance-based tasks completed by the patient during hospital visits. These are prone to bias and may not reflect motor abilities in real-world settings. Digital tools, such as wearable devices and other remote sensors, provide the opportunity for continuous, objective, and sensitive measurements of functional ability during daily life. Maintaining ambulation is of key importance to individuals with DMD. Stride velocity 95th centile (SV95C) is the first wearable acquired digital endpoint to receive qualification from the European Medicines Agency (EMA) to quantify the ambulation ability of ambulant DMD patients aged ≥5 years in drug therapeutic studies; it is also currently under review for the US Food and Drug Administration (FDA) qualification. <b><i>Summary:</i></b> Focusing on SV95C as a key example, we describe perspectives of multiple stakeholders on the promise of novel digital endpoints in neuromuscular disease drug development.

The evolution of patient-focused drug development and Duchenne muscular dystrophy

2020 ◽  
Vol 20 (1) ◽  
pp. 57-68
Author(s):  
Norah L. Crossnohere ◽  
Ryan Fischer ◽  
Emily Crossley ◽  
Elizabeth Vroom ◽  
John FP Bridges
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Drug Development ◽  
Patient Focused Drug Development

Drug development of vamorolone for Duchenne muscular dystrophy

2017 ◽  
Vol 27 ◽  
pp. S217-S218
Author(s):  
P. Clemens ◽  
M. Guglieri ◽  
L. Morgenroth ◽  
J. Damsker ◽  
A. Smith ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Drug Development

PRO38 Twenty Years of Clinical Trials in Duchenne Muscular Dystrophy: A Low Clinical Drug Development Success

2021 ◽  
Vol 24 ◽  
pp. S204
Author(s):  
A. Dovari ◽  
B. Inuganti ◽  
J. Nadimpally ◽  
S.M. Vatturi ◽  
R. Hyderboini ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Drug Development ◽  
Clinical Drug Development ◽  
Clinical Drug

Vision DMD: A drug development program for vamorolone in Duchenne muscular dystrophy

2017 ◽  
Vol 27 ◽  
pp. S17
Author(s):  
M. Guglieri ◽  
P. Clemens ◽  
A. Cnaan ◽  
J. Damsker ◽  
H. Gordish-Dressman ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Drug Development ◽  
Development Program ◽  
Drug Development Program

Vision DMD: Vamorolone (VBP15) drug development program for Duchenne muscular dystrophy

2017 ◽  
Vol 21 ◽  
pp. e238
Author(s):  
M. Guglieri ◽  
P. Clemens ◽  
A. Cnaan ◽  
J. Damsker ◽  
A. Arrieta ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Drug Development ◽  
Development Program ◽  
Drug Development Program

Vision DMD: Vamorolone drug development program for Duchenne muscular dystrophy

2016 ◽  
Vol 26 ◽  
pp. S156
Author(s):  
M. Guglieri ◽  
P. Clemens ◽  
A. Cnaan ◽  
J. Damsker ◽  
H. Gordish-Dressman ◽  
...  
Keyword(s):  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Drug Development ◽  
Development Program ◽  
Drug Development Program
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