Therapies for Inherited Retinal Dystrophies: What is Enough?
•IRDs affect over 4M people worldwide, leading to blindness and high societal cost•Despite a diversity of therapeutic approaches, many CTs have been abruptly halted•This raises key questions, e.g. what is a meaningful impact on patients’ daily life?•·Identifying reliable outcome measures for all act...
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| Published in: | Drug discovery today Vol. 29; no. 9; p. 104095 |
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| Main Authors: | , , , , |
| Format: | Journal Article |
| Language: | English |
| Published: |
England
Elsevier Ltd
01-09-2024
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| Online Access: | Get full text |
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| Summary: | •IRDs affect over 4M people worldwide, leading to blindness and high societal cost•Despite a diversity of therapeutic approaches, many CTs have been abruptly halted•This raises key questions, e.g. what is a meaningful impact on patients’ daily life?•·Identifying reliable outcome measures for all actors, including patients, is urgent•Rare disease CTs need specifically designed studies and global regulatory pathways |
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| Bibliography: | SourceType-Scholarly Journals-1 content type line 23 ObjectType-Editorial-2 ObjectType-Commentary-1 |
| ISSN: | 1359-6446 1878-5832 1878-5832 |
| DOI: | 10.1016/j.drudis.2024.104095 |