PONE-D-20-00658

FDA orphan drug designations for lysosomal storage disorders - a cross sectional analysis

PLOS ONE

Dear Dr Ries,

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PLOS ONE

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Reviewers' comments:

Reviewer's Responses to Questions

Comments to the Author

1. Is the manuscript technically sound, and do the data support the conclusions?

The manuscript must describe a technically sound piece of scientific research with data that supports the conclusions. Experiments must have been conducted rigorously, with appropriate controls, replication, and sample sizes. The conclusions must be drawn appropriately based on the data presented.

Reviewer #1: Yes

Reviewer #2: Partly

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2. Has the statistical analysis been performed appropriately and rigorously?

Reviewer #1: Yes

Reviewer #2: I Don't Know

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3. Have the authors made all data underlying the findings in their manuscript fully available?

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Reviewer #1: Yes

Reviewer #2: Yes

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4. Is the manuscript presented in an intelligible fashion and written in standard English?

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Reviewer #1: Yes

Reviewer #2: No

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5. Review Comments to the Author

Please use the space provided to explain your answers to the questions above. You may also include additional comments for the author, including concerns about dual publication, research ethics, or publication ethics. (Please upload your review as an attachment if it exceeds 20,000 characters)

Reviewer #1: The review explains the state of the art of development of treatments for LSDs. It is well written and with complete information.

The manuscript is well organized and clearly written.

I would suggest to change the figures, the points alone in them are difficult to understand. May be if you put a line, it could be better understandable.

Reviewer #2: The manuscript by Garbade et al, entitled "FDA orphan drug designations for lysosomal storage disorders - a cross sectional analysis" represents a useful summary and analysis of all orphan drug designation related to lysosomal storage disorders, compared to all orphan drug designations in the same period of time.

Although well-written, the manuscript presents a strange form, since Authors, instead of describing their results citing related Figures and Tables, as commonly performed, here present their results mainly using the Figure and Table legends, which renders results very difficult to read. Therefore, presentation of results should be totally re-written. In addition, Authors should provide all Figure and Table legends in a section separated from the main text. In its present form, the paper is really hard to understand and quite confusing.

The statistical analysis performed, although described in the methods section of the manuscript, is not clearly described in relation to the specific groups of data analyzed.

The name of the last Figure reported is Figure 1B instead of Figure 3B, please correct.

In addition, few English mistakes are present in the manuscript, please check and correct.

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Reviewer #1: No

Reviewer #2: No

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FDA orphan drug designations for lysosomal storage disorders – a cross-sectional analysis

PONE-D-20-00658R1

Dear Dr. Ries,

We are pleased to inform you that your manuscript has been judged scientifically suitable for publication and will be formally accepted for publication once it complies with all outstanding technical requirements.

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With kind regards,

Israel Silman

Academic Editor

PLOS ONE

Additional Editor Comments (optional):

Reviewers' comments: