Caregiver-perceived behavioral challenges in fragile X syndrome and implications for measuring treatment benefit in clinical trials.
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| Title: | Caregiver-perceived behavioral challenges in fragile X syndrome and implications for measuring treatment benefit in clinical trials. |
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| Authors: | Merikle E; Harmony Biosciences, Plymouth Meeting, PA, USA. emerikle@harmonybiosciences.com., Tich N; Harmony Biosciences, Plymouth Meeting, PA, USA., Sebree T; 1323 Consulting, LLC, Johnson City, TX, USA., Hagerman R; Medical Investigation of Neurodevelopmental Disorders (MIND) Institute, University of California Davis Medical Center, Sacramento, CA, USA.; Department of Pediatrics, University of California Davis School of Medicine, Sacramento, CA, USA., O'Quinn S; Perissos Inc, Wake Forest, NC, USA., Nomikos G; Harmony Biosciences, Plymouth Meeting, PA, USA., Bzdek K; Harmony Biosciences, Plymouth Meeting, PA, USA. |
| Source: | Journal of patient-reported outcomes [J Patient Rep Outcomes] 2026 Apr 01; Vol. 10 (1). Date of Electronic Publication: 2026 Apr 01. |
| Publication Type: | Journal Article |
| Journal Info: | Publisher: Springer International Publishing Country of Publication: Germany NLM ID: 101722688 Publication Model: Electronic Cited Medium: Internet ISSN: 2509-8020 (Electronic) Linking ISSN: 25098020 NLM ISO Abbreviation: J Patient Rep Outcomes Subsets: MEDLINE |
| Database: | MEDLINE Ultimate |
| ISSN: | 2509-8020 |
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| DOI: | 10.1186/s41687-026-01054-9 |
