Interim TEASE-3 Trial Data in Stargardt Disease with Michael B. Gorin, MD, PhD скачать в хорошем качестве

Interim TEASE-3 Trial Data in Stargardt Disease with Michael B. Gorin, MD, PhD

Positive interim data from the TEASE-3 clinical trial showed gildeuretinol (ALK-001) slowed the progression of Stargardt disease, the most common form of inherited macular degeneration in children and young adults, for up to six years. The first 3 patients treated with oral gildeuretinol in TEASE-3 have remained asymptomatic and free from disease progression over their treatment duration, ranging between 2 years and 6 years. In an interview with HCPLive, Michael B. Gorin, MD, PhD, of the departments of ophthalmology and human genetics at the David Geffen School of Medicine, UCLA, indicated that without gildeuretinol treatment, these patients were projected to experience vision loss within 2 years. “We could find evidence of early Stargardt changes with microperimetry or imaging, but they were completely asymptomatic. From a vision standpoint, they were functioning completely normally,” Gorin told HCPLive. “Those children who have been on this drug have managed to maintain a high level of vision and have developed almost no atrophy at all.” Interview Highlights 0:00 Introduction 0:09 Background on Stargardt disease, TEASE trials 7:34 Interim data from TEASE-3 trial 14:02 Next steps for TEASE-3 16:03 Implications of Gildeuretinol for patients with Stargardt disease Link to full article: https://www.hcplive.com/view/michael-... Other ophthalmology content: https://www.hcplive.com/clinical/opht... #ophthalmology #rarediseases