Nearly Three Years Later, LUXTURNA®™ Treatments Continue to See Success
Since its launch in March 2018, breakthrough gene therapy LUXTURNA®™ continues to be successful in helping improve vision in people with...
Research Updates
All About Clinical Trials
Clinical trials are never done in a vacuum, or in a medieval basement where Dr. Frederick Frankenstein (pronounced Fronkensteen), his pretty...
Retinal Disease Gene Therapy Breakthroughs Trace Their Roots to 19th Century Research
Theodor Karl Gustav von Leber would be proud. So would Adolphe Franceschetti and Carl-Henry Alström. Their research from the 19th and 20th centuries laid the foundation...
Dr. Jean Bennett: ‘Seeing the Light with Retinal Gene Therapy’
Known as a pioneer in gene therapy, Jean Bennett, MD, PhD, surveyed her audience of patients and families living with Leber congenital amaurosis and...
Explosive Growth Seen in Field of Rare Inherited Retinal Disease Research
Advances in genetic sequencing boosted research into rare inherited retinal diseases (IRDs), making a tremendous impact on the number of clinical trials...
Diagnosis to Treatment: Pioneering LCA Patient Eases the Journey
As a global advocacy organization dedicated to helping those affected by blindness caused by rare inherited retinal disease, Hope in Focus...
The Road to Treatment: Understanding How Therapies Are Developed
Successful clinical drug trials are a cornerstone of U.S. Food and Drug Administration approval, such as with LUXTURNA™, a ground-breaking genetic therapy that...
IRD Milestones: Reasons to Be Excited
1971 – Just those numbers in white on a black page appeared on the big screen. That’s how Brian Mansfield, PhD.,...
New Patient Services Program Helps People Navigate Their Road To A Cure
Now that LUXTURNA™ has come to market as a revolutionary vision-restoring genetic treatment, how does it get to patients? The answer to...
Making Connections: Nightstar Therapeutics
I was so happy to recently speak with Samantha Vieira, Senior Director of Program Management of Nightstar Therapeutics. Nightstar is a...