Molecular and Biochemical Therapeutic Strategies for Duchenne Muscular Dystrophy

Round 1

Reviewer 1 Report

Comments and Suggestions for Authors

The authors provide an insightful review of innovative therapies for Duchenne muscular dystrophy, contributing valuable knowledge to the field. However, there are areas that need improvement:

 

The introduction lacks a clinical definition of the disease and does not describe phenotypes derived from pathogenic variants in the DMD gene, such as BMD and dilated cardiomyopathy 3B.

The introduction lacks references and contains common and irrelevant information, such as stating that the DMD gene is the largest without providing a reference or explaining its relevance.

The use of clinical terminology is imprecise, and the images included in the paper are of low resolution and poor quality.

Some paragraphs lack logical coherence, such as discussing the importance of blocking exon 51 as a therapeutic strategy when mentioning that exon 51 deletions are the most common mutatio which may confuse readers (if exon 51 is deleted you cannot skip it).

The title does not accurately reflect the content of the review, as the focus is on the biochemistry and molecular biology of therapeutic strategies rather than the disease management typically discussed in clinical guidelines.

I recommend that the authors include a paragraph on the signs and symptoms of DMD, describe clinical phenotypes, and add references to support the information provided in the introduction.

Maybe consider changing the title

 

 

Comments on the Quality of English Language

You could use a more formal english for this review, there are common expressions that are not optimal for a scientific text

Author Response

Please see the attachment

Author Response File: Author Response.pdf

Reviewer 2 Report

Comments and Suggestions for Authors

The paper describes the existing therapies as well as those under research for Duchenne Muscular Dystrophy.

I feel the review is too lengthy and needs focusing on either genetic therapies or other therapies.

The aim of the review is to 'manage Duchenne Muscular Dystrophy'; genetic techniques are described in detail but there is a lack of focus on clinical (patient-based) evidence/effectiveness for most of genetic strategies mentioned. 

Additionnally, many statements lack referencing.

 

Author Response

Please see the attachment

Author Response File: Author Response.pdf

Round 2

Reviewer 1 Report

Comments and Suggestions for Authors

Good job, this title is more appropriate for the content of the manuscript

Reviewer 2 Report

Comments and Suggestions for Authors

The reading of the manuscript has improved after changes made by the authors.