Using a new digital format, the EFF welcomed over 50 stakeholders to discuss continued clinical development in Leber congenital amaurosis 10 (LCA10) and the importance of both the expert clinical and patient perspectives in research.
Clinical development for Leber congenital amaurosis 10
Opening the virtual EFF, Daniel de Boer, ProQR Founder and CEO, welcomed all attendees and provided an update on the continued clinical development mission ProQR is striving toward, even in these unprecedented times. Audience members then heard from Aniz Girach, MD, ProQR Chief Medical Officer, who outlined the latest research findings from the ProQR sepofarsen program for LCA10.
Following the presentation, Dr Girach was joined by Dr. Ian M. MacDonald, a leading expert in the inherited retinal diseases (IRD) field. Throughout the IRD Expert Perspective session, webinar participants examined the latest LCA10 findings and what they mean for IRD clinical development and research, as well as the impact on the patient community.
Watch the Expert Perspective video call with Dr. Aniz Girach and Dr. Ian MacDonald on LCA and the latest clinical data for sepofarsen.
Live questions and answers session
Following the experts’ discussion, the EFF then dove into a live question and answer session with the panel consisting of ProQR Patient & Caregiver Steering Committee Member Francesca Diodati, Ian MacDonald and Aniz Girach. This gave the audience a chance to ask the panelists questions about the patients’ perspective on clinical research.
Watch the ProQR talks video call “Why is the patient perspective in research important?” with Francesca Diodati and Laura Brady.
Webinar recording
The full webinar is available for replay with English captions on our YouTube channel, where you can navigate through the video using the links in the description.