Since its launch in March 2018, breakthrough gene therapy LUXTURNA®™ continues to be successful in helping improve vision in people with inherited retinal disease due to mutations in both copies [...]
Known as a pioneer in gene therapy, Jean Bennett, MD, PhD, surveyed her audience of patients and families living with Leber congenital amaurosis and declared: “YOU are all the pioneers!” […]
As a global advocacy organization dedicated to helping those affected by blindness caused by rare inherited retinal disease, Sofia Sees Hope connects families with Leber congenital amaurosis [...]
It’s you. It’s all about you. The journey from identifying a rare disease, to conducting studies, to approving a treatment, is long – but it always starts with the patient, […]
Transforming laboratory research into real-life therapy for patients is a rare occurrence. But when it does happen, it’s big. Huge, in fact. Ask Dr. Audina M. Berrocal, the pediatric retinal […]
This is the seventh in a series following the progress of Creed Pettit, a 9-year-old Florida third-grader, who completed treatment in March with the breakthrough gene-therapy drug called [...]
A revolutionary genetic treatment could improve the vision of 5-year-old Maverick Johnston, but his mom first wants to know more about the side effects and capabilities of the breakthrough drug [...]
This is the sixth in a series following the progress of Creed Pettit, a 9-year-old Florida third-grader, who completed treatment in March with the breakthrough gene-therapy drug called LUXTURNA™, [...]
Now that LUXTURNA™ has come to market as a revolutionary vision-restoring genetic treatment, how does it get to patients? The answer to that question and many others can be found within […]
This is the fifth in a series following the progress of Creed Pettit, a 9-year-old Florida third-grader, who completed treatment in March with the breakthrough gene-therapy drug called LUXTURNA™, [...]