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bluebird bio (Germany) GmbH Josephspitalstr. 15 | Regus 80331 München, Germany http://www.bluebirdbio.com
Contact Ms Ingrid Goldberg +1 410-960-5022
Company logo of bluebird bio (Germany) GmbH
bluebird bio (Germany) GmbH

bluebird bio präsentiert neue Ergebnisse aus dem klinischen Entwicklungsprogramm für die Gentherapie mit elivaldogene autotemcel (eli-cel, Lenti-D™) zur Behandlung der zerebralen Adrenoleukodystrophie (CALD), einschließlich aktualisierter Langzeitdaten

(PresseBox) (Cambridge, Mass./USA, )
.
- Die Langzeitergebnisse der Phase-2/3-Studie Starbeam (ALD-102/LTF-304) weisen auf ein dauerhaftes Ansprechen nach der Behandlung mit eli-cel hin. Alle 20 Patienten, die nach zwei Jahren frei von schweren funktionellen Behinderungen (major functional disabilities, MFDs*) waren (von 23 auswertbaren Patienten), blieben bis zur letzten verfügbaren Nachbeobachtung MFD-frei. Dies trifft ebenfalls für alle 10 Patienten zu, die mindestens das fünfte Jahr der Nachuntersuchung erreicht haben.1
- 31 von 32 Patienten der Studie ALD-102 hatten nach der Behandlung mit eli-cel stabile neurologische Funktionswerte (NFS), darunter 24 Patienten mit einem Wert von Null bei der letzten Untersuchung.1
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