{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,14]],"date-time":"2026-10-14T22:19:30Z","timestamp":1792016370988,"version":"4.4.0"},"reference-count":38,"publisher":"Georg Thieme Verlag KG","issue":"03","content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Klin Monbl Augenheilkd"],"published-print":{"date-parts":[[2025,3]]},"abstract":"<jats:title>Zusammenfassung<\/jats:title><jats:p>\n          ABCA4-assoziierte Netzhautdystrophien, einschlie\u00dflich des von Stargardt erstmals beschriebenen Morbus Stargardt umfassen eine heterogene Gruppe erblicher Netzhauterkrankungen, die durch Mutationen im ABCA4-Gen verursacht werden und rezessiv vererbt werden. Diese Erkrankungen f\u00fchren aufgrund einer fortschreitenden Degeneration von Photorezeptoren und retinalem Pigmentepithel zu einem Sehverlust, f\u00fcr den es aktuell keine zugelassene Therapie gibt. Aufgrund der Fortschritte in den letzten Jahren und der gut verstandenen Pathobiologie existieren bereits vielversprechende Therapieans\u00e4tze in der klinischen Entwicklungsphase, wobei pharmakologische Ans\u00e4tze zu den am weitesten fortgeschrittenen Therapieoptionen geh\u00f6ren. Pr\u00e4klinische und klinische Studien zeigen Fortschritte bei der Entwicklung von Medikamenten, die das Potenzial haben, die Progression dieser Erkrankungen deutlich zu verlangsamen. Darunter befinden sich Wirkstoffe, die die Lipofuszin-Akkumulation in der\n          Netzhaut verringern k\u00f6nnen, die Bildung toxischer Vitamin-A-Dimere verlangsamen oder Lipofuszin aus der Netzhaut entfernen k\u00f6nnen. Weitere Substanzen greifen in den Vitamin-A-Stoffwechsel im Sehzyklus ein, indem sie die verf\u00fcgbare Menge an Vitamin A im Auge reduzieren, was zu einer geringeren Bildung toxischer Stoffwechselnebenprodukte f\u00fchren sollte. Dieser Beitrag fasst die der ABCA4-assoziierten Netzhautdegeneration zugrunde liegenden Pathophysiologie zusammen und erl\u00e4utert aktuelle pharmakologische Therapieans\u00e4tze.<\/jats:p>","DOI":"10.1055\/a-2533-1009","type":"journal-article","created":{"date-parts":[[2025,3,24]],"date-time":"2025-03-24T23:40:23Z","timestamp":1742859623000},"page":"205-212","source":"Crossref","is-referenced-by-count":1,"title":["Pharmakotherapie von ABCA4-assoziierten Netzhautdystrophien"],"prefix":"10.1055","volume":"242","author":[{"given":"Maximilian","family":"Gerhardt","sequence":"additional","affiliation":[{"name":"Augenklinik der LMU, Klinikum der Universit\u00e4t M\u00fcnchen, Deutschland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Katarina","family":"Stingl","sequence":"additional","affiliation":[{"name":"Universit\u00e4ts-Augenklinik, Department f\u00fcr Augenheilkunde, Universit\u00e4t T\u00fcbingen, T\u00fcbingen, Deutschland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Milda","family":"Reith","sequence":"additional","affiliation":[{"name":"Universit\u00e4ts-Augenklinik, Department f\u00fcr Augenheilkunde, Universit\u00e4t T\u00fcbingen, T\u00fcbingen, Deutschland"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"194","published-online":{"date-parts":[[2025,3,24]]},"reference":[{"key":"ref1","doi-asserted-by":"publisher","first-page":"534","DOI":"10.1007\/BF01961301","article-title":"\u00dcber famili\u00e4re, progressive Degeneration in der Maculagegend des Auges","volume":"71","author":"K Stargardt","year":"1909","journal-title":"Albrecht von Graefes Archiv f\u00fcr Ophthalmologie"},{"key":"ref2","doi-asserted-by":"publisher","first-page":"236","DOI":"10.1038\/ng0397-236","article-title":"A photoreceptor cell-specific ATP-binding transporter gene (ABCR) is mutated in recessive Stargardt macular dystrophy","volume":"15","author":"R 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