Moms are often the chief medical officers of their households and the first line of defense when it comes to their family’s health. They are on call 24 hours, through every stage of life, and most of it goes unacknowledged because they make it look so easy. To mothers of all kinds, thank you for doing the hardest job in the world. Thank you for working tirelessly to care for others day after day, for never giving up on those who look up to you, and for understanding the powerful connection a mother figure can have in a person’s life. Happy Mother’s Day from all of us at Lehigh Valley Center For Sight.
Editas Medicine , a company developing gene-editing treatments, has received authorization from the US Food and Drug Administration to launch a clinical trial for its emerging CRISPR/Cas9 therapy for people with a mutation in the gene CEP290, which causes Leber congenital amaurosis 10 (LCA10). LCA causes severe vision loss or blindness at birth. Known as EDIT-101, the treatment will be evaluated in a Phase 1/2, dose-escalation clinical trial. The company expects to enroll 10 to 20 patients. Further details and contact information for the study will be listed soon at www.clinicaltrials.gov . EDIT-101 targets a specific mutation, “c.2991+1655A>G” in intron 26, of the CEP290 gene. Editas has earned a $25 million milestone payment from Allergan as part of a drug-development alliance. “We are very pleased with this historic milestone in the treatment of inherited retinal diseases,” says Stephen Rose, PhD, chief scientific officer, Foundation Fighting Blindness. “EDIT-101 is the fi...
As the holidays approach, there are many digital gadgets children may be asking for or receiving. Some of the more popular gifts include flat-screen TVs, tablets, phones, streaming devices (Firestick, Roku, Apple TV, etc.), and gaming systems. Even Fisher-Price is offering the Think & Learn Smart Cycle, which syncs with a tablet for kids to play learning games while they ride the attached stationary bike. While some of these devices add learning opportunities, most of them come with added screen time, which may be cause for concern. The effects of excessive screen time are usually considered when adults work on computers for 8+ hours a day , but parents are starting to notice their children’s symptoms. Device Use & Symptoms in Young People In a survey of 300 US parents, half of the participants reported that their children spend too much time on devices. However, over two-thirds believe device usage won’t change and say they won’t be cutting back any time soon. Because of thi...
Applied Genetic Technologies Corporation (AGTC) , a leading developer of gene therapies, has reported the six-month follow-up data in all patients treated with its gene therapy for X-linked retinoschisis (XLRS) in its Phase 1/2 clinical trial. The company reported that no consistently significant clinical activity from the treatment was observed after six months. AGTC will continue to analyze all of the XLRS trial data and complete all visits specified in the clinical protocol. However, if the data at additional follow-up data visits are consistent with the current six-month findings, the company has stated that they will not further develop the product. A total of 27 subjects were treated, and all completed study visits through at least six months. Thirteen adult participants received the high dose of the treatment. The study subjects also included five pediatric patients who received the medium dose, and nine adults who received medium and low doses of the gene therapy. All partici...
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