Living with adrenoleukodystrophy: adult patient and caregiver perspectives
Abstract Introduction Adrenoleukodystrophy (ALD) is a rare, X-linked disease caused by pathogenic ABCD1 gene variants, resulting in heterogeneous and debilitating conditions. We report on an Externally-Led Patient-Focused Drug Development (EL-PFDD) meeting involving patients and caregivers, Food and...
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| Главные авторы: | , , , , , , |
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| Формат: | Artigo |
| Язык: | Inglês |
| Опубликовано: |
BMC
2026-01-01
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| Серии: | Orphanet Journal of Rare Diseases |
| Предметы: | |
| Online-ссылка: | https://doi.org/10.1186/s13023-025-04130-3 |
| Метки: |
Нет меток, Требуется 1-ая метка записи!
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