Month: February 2019

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The Assistance Fund, an independent charitable patient assistance foundation that helps patients and families facing high medical out-of-pocket costs, today launched a new program that offers financial support for individuals with inherited retinal diseases. The fund will assist patients in paying out-of-pocket medical costs incurred as a result of their condition, including health insurance premiums, copayments and incidental medical expenses.

Ophthotech Corporation (NASDAQ: OPHT) announced today that the first patient has been enrolled in the Phase 2b randomized, double masked, sham controlled clinical trial assessing the efficacy and safety of Zimura® (avacincaptad pegol), complement C5 inhibitor, in patients with autosomal recessive Stargardt disease (STGD1). STGD1 is an inherited orphan retinal disease causing vision loss during childhood or adolescence. Currently there are no FDA or EMA approved treatment options available for patients with STGD1. Over 30 sites have been identified for this clinical trial, making it one of the largest interventional clinical trials for STGD1 to date. The scientific details of the Stargardt clinical trial will be presented at the 2018 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Honolulu, Hawaii.

Key Updates

A Symposium in honor of Dr. Gustavo Aguirre, Recipient of the 2017 Proctor Medal, Association for Research in Vision and Ophthalmology.

GAINESVILLE, Fla., and CAMBRIDGE, Mass., Aug. 10, 2017 (GLOBE NEWSWIRE) — Applied Genetic Technologies Corporation (NASDAQ:AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare diseases, today announced that it has filed an Investigational New Drug application (IND) with the U.S. Food and Drug Administration to conduct a Phase 1/2 clinical trial of the company’s gene therapy product candidate for the treatment of X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene.