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Therapeutic opportunities and clinical outcome measures in Duchenne muscular dystrophy.


ABSTRACT:

Introduction

Duchenne muscular dystrophy (DMD) is a devastatingly severe genetic muscle disease characterized by childhood-onset muscle weakness, leading to loss of motor function and premature death due to respiratory and cardiac insufficiency.

Discussion

In the following three and half decades, DMD kept its paradigmatic role in the field of muscle diseases, with first systematic description of disease progression with ad hoc outcome measures and the first attempts at correcting the disease-causing gene defect by several molecular targets. Clinical trials are critical for developing and evaluating new treatments for DMD.

Conclusions

In the last 20 years, research efforts converged in characterization of the disease mechanism and development of therapeutic strategies. Same effort needs to be dedicated to the development of outcome measures able to capture clinical benefit in clinical trials.

SUBMITTER: Ricci G 

PROVIDER: S-EPMC9126754 | biostudies-literature | 2022 Dec

REPOSITORIES: biostudies-literature

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Therapeutic opportunities and clinical outcome measures in Duchenne muscular dystrophy.

Ricci Giulia G   Bello Luca L   Torri Francesca F   Schirinzi Erika E   Pegoraro Elena E   Siciliano Gabriele G  

Neurological sciences : official journal of the Italian Neurological Society and of the Italian Society of Clinical Neurophysiology 20220524 Suppl 2


<h4>Introduction</h4>Duchenne muscular dystrophy (DMD) is a devastatingly severe genetic muscle disease characterized by childhood-onset muscle weakness, leading to loss of motor function and premature death due to respiratory and cardiac insufficiency.<h4>Discussion</h4>In the following three and half decades, DMD kept its paradigmatic role in the field of muscle diseases, with first systematic description of disease progression with ad hoc outcome measures and the first attempts at correcting  ...[more]

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