Exploring barriers to clinical trial readiness among the myotonic dystrophy community: a mixed-methods study
Julia Stellmann Wrenn, Ali Ryan-Mosley, Jonah Watt, Erin Hellthaler, Tanya Stevenson, Andy Rohrwasser · 2026
Read Exploring barriers to clinical trial readiness among the myotonic dystrophy community: a mixed-methods study by Julia Stellmann Wrenn, Ali Ryan-Mosley, Jonah Watt, Erin Hellthaler, Tanya Stevenson, Andy Rohrwasser (2026) in the Tower of Babel, a free online library of public-domain works preserved in clean, searchable text. No sign-up. The full text is served as a plain-text file at a predictable URL, where text is present: https://antarctic-labs.com/tower-of-babel/PMC13602584.txt.
Description
Julia Stellmann Wrenn leads a six-author team mapping in 2026 why drug trials for myotonic dystrophy struggle to get ready. This inherited, whole-body disorder varies hugely between patients, which tangles trial design. The team gathered views from every side: focus groups, surveys, and interviews with 65 pharmaceutical partners, 35 patients and caregivers, 12 lead investigators, and 12 research coordinators. Qualitative themes were then blended with survey numbers.
Three barriers came up everywhere. Trials lack validated outcome measures that capture what patients feel matters. Companies share data poorly and guard natural history information, which smaller firms often cannot afford. Trial designs also burden participants with cost, travel, and access problems. Patients flagged swallowing trouble, fatigue, and cognitive impairment as debilitating yet rarely prioritized. Proposed fixes include a joint session with the US Food and Drug Administration on patient-centered endpoints, a coordinated registry, better site preparation, and lower-burden designs led with advocacy groups.
Catalog record
| CREATOR | Julia Stellmann Wrenn, Ali Ryan-Mosley, Jonah Watt, Erin Hellthaler, Tanya Stevenson, Andy Rohrwasser |
|---|---|
| YEAR | 2026 |
| COLLECTION | PAPERS |
| FORMAT | TXT |
| FILE SIZE | 80611 |
| LICENSE | https://creativecommons.org/publicdomain/zero/1.0 |
| RIGHTS | CC0 |
| SOURCE | Europe PMC |