Foundation Fighting Blindness (FFB) are to invite a “Patient-Focused Drug Development” meeting, in the coming months, with Food and Drug Administration (FDA) representatives. The objective of the meeting is to better understand the impact of vision loss on activities of daily living and perspectives on the risks and benefits of potential treatments. The charity organisation will support a number of companies (Applied Genetic Technologies Corporation (AGTC) and Biogen) to collaborate with an open valuable platform: “My Retina Tracker Registry” (MRTR). The initiative is to seeking ~300 males, 18-years of age and older, with XLRP, to be registered in MRTR, and who live in the US. The participant must have a mutation in the gene RPGR identified as the cause of XLRP and approximately 10-20% of the survey participants will include caregivers of people with XLRP.
According to FFB, the survey should take about 25 minutes to complete and is designed to be accessible to people with low vision and those who use computer screen readers. Participation in this survey will not impact your ability to take part in any other research programs, surveys, or clinical trials. Commenting on the initiative, Todd Durham, PhD, Senior Vice-President, for Clinical & Outcomes Research, Foundation Fighting Blindness, stated that: “[t]he survey is an excellent opportunity for people with XLRP and their caregivers to share their expectations of the benefits of emerging therapies. The information collected helps all partners involved in therapy development to gain a better understanding of the role of vision on activities of daily living and understand the needs of the targeted population.”
The My Retina Tracker Registry is an open-access, no-cost genetic testing programme to populate a valuable research database of participants and families affected by rare inherited retinal degenerative diseases (IRD). There are over 20 retinal degenerative diseases in the database including retinitis pigmentosa, Leber congenital amaurosis, Stargardt disease, Usher syndrome, Best disease, choroideremia, and achromatopsia. The FFB has explained that the registry is designed to share de-identified information within the IRD research and clinical communities about participants with an inherited retinal disease to help accelerate the discovery of treatments and cures. Using the data, the registry aims to understand how common each type of retinal disease is, “how it impacts people’s lives, how the disease progresses, the genes that cause the disease, and helps researchers and companies to efficiently find people who might be interested in participating in research studies and clinical trials”.