Clinical trials for neurodevelopmental disorders: At a therapeutic frontier
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A well-powered clinical trial that failed to replicate promising results in animal models of fragile X syndrome yields important lessons for clinical trial design (Berry-Kravis et al ., this issue).
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In some cases, knowledge of the underlying pathophysiology seems sufficient for the development of novel therapies, yet results are disappointing when new drugs are tested in adult patients.
“In some cases, however, our knowledge of the underlying pathophysiology seems sufficient for the development of novel therapies, yet results are disappointing when new drugs are tested in adult patients 4 .”
From Developmental timing and critical windows for the treatment of psychiatric disorders (2016) · cites this paper as [4]
Discoveries are not easily translatable to humans.
“Yet, discoveries are not easily translatable to humans 4 .”
From Developmental timing and critical windows for the treatment of psychiatric disorders (2016) · cites this paper as [4]
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Cites
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- Authors
- Jeste, S. S., & Geschwind, D. H.
- Affiliations
- University of California, Los Angeles (US)
- Corresponding author
- Geschwind, Daniel H.
- Venue
- Science Translational Medicine
- Published
- 2016-01-13
- Volume
- 8
- Issue
- 321
- Pages
- 321fs1-321fs1
- ISSN
- 1946-6234 (ISSN-L)
- 1946-6242
- OpenAlex
- W2237434427
- Cited by
- 51
- References
- 9
- Citations by year
- 2025: 2 · 2024: 2 · 2023: 3 · 2022: 5 · 2021: 8 · 2020: 5 · 2019: 3 · 2018: 6 · 2017: 7 · 2016: 9 · 2015: 1
- FWCI
- 6.73
- Type
- journal article
- Language
- en
- Open access
- green
- escholarship.org
- Locations
- Science Translational Medicine (publishedVersion)
- PubMed (publishedVersion)
- eScholarship (California Digital Library) (submittedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genetics and Neurodevelopmental Disorders
- Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
- Health Sciences › Medicine › Public Health, Environmental and Occupational Health › Ethics in Clinical Research
- Keywords
- neurodevelopmental disorders, fragile X syndrome, clinical trial design, translational research, animal models, replication failure, treatment development
- MeSH terms
- Animals, Fragile X Syndrome, Humans, Fragile X Mental Retardation Protein, Fragile X Messenger Ribonucleoprotein 1, Neurodevelopmental Disorders
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