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Digital Supplement | Sponsored by Novartis Pharma AG

Starting With the End in Mind: Understanding the Patient Experience

To watch the presentation by Dr. Bart P. Leroy at EURETINA, please click on the link at the end of this article.

This supplement is intended for non-promotional scientific purposes only and may contain information on products or indications currently under investigation and/or that have not been approved by the regulatory authorities. The opinions and views expressed in this supplement are those of the faculty members and do not necessarily constitute the opinions or recommendations of Novartis. The presentations were accurate at the time of presentation. Any data on competitor product(s) are based on publicly available information at the time of presentation. Prescribing information may vary depending on local health authority approval in each country. Before prescribing any product, always refer to the SmPC or product information approved in your local country. Permissions for all content within have been received from each copyright holder for the presentations. Separate use, adaptation, and/or translation requires application for specific use permissions from each copyright holder. All product names, trademarks and registered trademarks are property of their respective owners.

The first pediatric patient in the world to receive voretigene neparvovec did so as a child as part of the phase 1 study, which took place in Philadelphia, PA, USA. Over 10 years later, and now a young adult studying psychology at university in Belgium, he spoke with Dr. Bart P. Leroy to share his experiences.

Bart P. Leroy, MD, PhD: Prior to receiving treatment, what symptoms did you experience living with an inherited retinal disease (IRD)?

Patient: It was a long time ago, so my memories are a bit limited, but my father tells a story of being on a family holiday in Turkey. In the bright sunlight of the daytime I was able to run around the swimming pool with my sister with little sign that I had any visual impairment. However, in the evenings when we went out to eat at a restaurant, I couldn’t see a thing on my plate and was basically blind. That’s something that I remember quite well—when I was inside, during winter, or when it was dark outside, I couldn’t see anything. I was basically completely night blind.

Dr. Leroy: What do you remember about the treatment itself?

Patient: I remember that after the surgery when all the patches were removed it was painful because of the light. The doctors wanted to look into my eye using a torch, which really hurt because I wasn’t used to that amount of light. Then, when we left Philadelphia, we flew out in the evening after dark. When the plane took off, I could see the lights of the city with my right eye—the eye that had received the treatment—but I wasn’t able to see them with my left eye. With my left eye it was completely black.

Dr. Leroy: How did this treatment impact what you do on a daily basis?

Patient: It has greatly affected my day-to-day life. Before the treatment, I worked completely in braille. After treatment, I started working on the computer with the aid of magnification. In my free time I use my vision to do things that I wouldn’t have been able to do without having treatment. In the week I live alone, and I’m more mobile now. My visual field is still not normal, so I do use a cane, but I use my cane in a different way than a blind person. I don’t follow walls or guidelines with my cane—I navigate around a city visually. I just use my cane for safety, in case of an object that I haven’t seen and also as a signal to others that I’m visually impaired.

Dr. Leroy: What message would you have for clinicians who may be seeing patients with IRDs such as yours?

Patient: My message would be that this is a really important treatment for two reasons. One is the visual improvement. Although my vision is not perfect, the improvement from what it was like previously makes a huge difference in day-to-day life. The second reason is something that, in my opinion, is underestimated, which is stability. I know that without treatment I would have been completely blind by now. Comparing the vision that I have today with the prospect of complete blindness makes me really value the stability of my vision.

Dr. Leroy: Your parents have previously described to me how they had to visit many different eye specialists before finally receiving your IRD diagnosis. What do you think about being referred as a patient to a super-specialist center?

Patient: It took my parents 7 or 8 years just to know the name of my disease and what caused it. If you are a doctor examining a patient, and you don’t know for sure what condition they have, I think referring them to someone who is a specialist in that area is one of the best things you can do.

To watch the presentation by Dr. Bart P. Leroy at EURETINA 2019, please click here.