Displaying 711–720 of 858 results
Dec 17, 2017
“I try not to get my hopes up too much, but I never lose hope; I’m determined. I just want to see my son’s face, see him get married, & see my grandchildren. I encourage everyone with a retinal disease to get a genetic test & to never ever give up hope.“
Nov 21, 2017
Stem-Cell Therapy Clinics Remain Inadequately Regulated, Pose Risk to Patients
If a clinic is charging for a stem-cell treatment or procedure for an IRD, it is probably not legit. The expense to the patient is a major red flag.
Nov 15, 2017
ProQR Doses First Participant in Its LCA10 Therapy Clinical Trial
The company plans to report interim, six-month study results in 2018 and 12-month results in 2019.
Oct 13, 2017
FDA Committee Unanimously Recommends Approval for Spark's RPE65 Gene Therapy - Final Decision Due in January 2018
An advisory committee comprised of FDA-selected experts voted unanimously – 16 to 0 – to recommend approval.
Oct 12, 2017
FDA Advisory Committee Unanimously Recommends Approval of Investigational LUXTURNA™ (voretigene neparvovec) for Patients with Biallelic RPE65-mediated Inherited Retinal Disease
Investigational LUXTURNA has the potential to be both the first pharmacologic treatment for an inherited retinal disease (IRD) and the first gene therapy for a genetic disease in the United States
First adeno-associated viral (AAV) vector investigational candidate to reach this stage of FDA review
Investigational LUXTURNA clinical program includes up to four years of efficacy data, with observation ongoing
Oct 9, 2017
Joe Caruso, founder of FOCUS Wine Foundation, knows life both with and without the ability to see. His first memory is sitting in the waiting room with his mother at the Scheie Eye Institute in Philadelphia.
Sep 27, 2017
The Foundation's Investments Are Filling the Pipeline for Vision-Saving Therapies
In addition to funding promising biotech start-ups, the Foundation Fighting Blindness has played a critical role in developing research talent.
Sep 20, 2017
Heather Presnar’s Story: Living with Stargardt Disease
When you’re the catcher, the ball always comes to you. Heather Presnar wouldn’t have had it any other way.
Aug 30, 2017
MeiraGTx Treats First Patient in XLRP Gene-Therapy Trial
The MeiraGTx gene therapy involves injection of healthy copies of RPGR underneath the retina. The RPGR copies are contained in a human-engineered virus — known as an adeno-associated virus or AAV — designed to readily penetrate retinal cells to deliver the therapeutic genetic cargo.
Aug 7, 2017
Sight and Song: The Christian Guardino Story
Christian Guardino has been singing for as long as he can remember; it’s the seeing that is something new.