August 19th, 2019: “EURETINA-Brief”© Issue No. 198
by Dr. Gearóid Tuohy
Dear EURETINA Members,
A very warm welcome to the August 19th, 2019 edition of EURETINA’s web-based digital magazine, “EURETINA Brief”. EURETINA are delighted to continue our delivery of up-to-date summary briefs on a range of topics of interest to retinal clinicians, specialists and researchers across Europe. This resource is designed to accommodate the very busy schedules of all our members by providing them with a short overview of some new developments and announcements in our field over recent weeks.
As in previous issues we have incorporated a feedback section where you can comment on any of the news items or articles under discussion and we very much welcome all contributions. Previous articles and issues can be found in the archive section on this website.
The current issue highlights a number of research activities, clinical / regulatory milestones, and market / business developments in our field, including research of genotype-phenotype correlations in Stargardt disease which highlight considerable complexity; clinical research for retinal gene therapy for X-linked retinoschisis show that AAV8 treatment had positive safety and tolerability outcomes, and; marketing activities reporting that UK researchers highlight of negative consequences of Brexit for the development of and access to medicines for orphan (rare) diseases in paediatrics.
Finally, our feature bio-ophthalmology article reports on the need for expert research scientists to support the evaluation of new drug approvals for novel technologies, including retinal stem cell and gene therapies. The pace of new technology and novel medical treatments has reached a point at which regulators and assessors require additional expert evaluation in reviewing and assessing marketing authorizations. Specialist researchers in fields such as genetics, stem cell biology, gene editing and others, are required to assist regulators assess and evaluate new therapies that rapidly progress from academic research to clinical application. Recent examples include on the submission of a marketing application to the European Medicines Agency for Investigational “LUXTURNA” (voretigene neparvovec). The application arises from several years research involving academic, regulatory and industry experts to devise a therapy to treat RPE-65 related retinopathies.
As always, your increased interaction within the EURETINA web community serves to expand your professional network and keep you up to date with the latest initiatives, activities and research in your field. Our hope is that such cross-fertilisation in an active web-based platform, including our LinkedIn page, will lead to increased collaborative opportunities and ultimately to improved patient care. All readers are invited to submit comments or responses to any of the stories featured and we look forward to hearing from you over the coming month.
Best wishes,
Dr. Gearóid Tuohy, EURETINA