Results 41 to 50 of about 1,367 (120)

Perspectives of people with inherited retinal diseases on ocular gene therapy in Australia: protocol for a national survey

BMJ Open, 2021
Introduction Voretigene neparvovec-rzyl (Luxturna) was approved by the Australian Therapeutic Goods Administration on 4 August 2020 for the treatment of biallelic mutations in the RPE65 gene, a rare cause of congenital and adult-onset retinal dystrophy ...
, Fred K Chen, Alex W Hewitt, Alan Ma, John Grigg, Keith R Martin, Robyn Jamieson, Heather G Mack, Fleur O’Hare, David Mackey, John De Roach, Alexis Ceecee Britten-Jones, Myra McGuinness, Nicole Tindill, Lauren Ayton, Anai Gonzalez Cordero, Thomas L Edwards, Gladys Ho, Michael Hogden, Anthony Kwan, Tina Lamey, Terri McLaren, Benjamin Nash, Jon Ruddle, Matthew Simunovic, Ingrid Sinnerbrink, Deepa Ajay Taranath, Jen Thompson, Jaclyn White   +28 more
doaj   +1 more source

Early experiences with voretigene neparvovec (Luxturna) gene therapy in Poland: an analysis of four patients with biallelic RPE65 gene mutations

Klinika Oczna
Marcin Stopa, Zuzanna Niedziela-Schwartz, Martyna Mieszczak, Piotr Rakowicz, Maciej Krawczyński   +4 more
doaj   +2 more sources

Retinitis Pigmentosa: Novel Therapeutic Targets and Drug Development

Pharmaceutics, 2023
Retinitis pigmentosa (RP) is a heterogeneous group of hereditary diseases characterized by progressive degeneration of retinal photoreceptors leading to progressive visual decline.
Kevin Y. Wu, Merve Kulbay, Dana Toameh, An Qi Xu, Ananda Kalevar, Simon D. Tran   +5 more
doaj   +1 more source

Evaluation of the conditional reimbursement of Voretigene Neparvovec (Luxturna) for inherited retinal dystrophies caused by RPE65 gene mutations in Norway: A value of information analysis.

, 2022
Background: Novartis submitted their health technology assessment in 2019 to the Norwegian Medicine Agency (NOMA), however, in 2020, NOMA discarded the submission by Novartis as they deemed Voretigene Neparvovec (Luxturna) to not be cost-effective ...
Nedberg, Christoffer
core  

Gene therapy in retinal diseases: A review

Indian Journal of Ophthalmology, 2021
Over 2 million people worldwide are suffering from gene-related retinal diseases, inherited or acquired, and over 270 genes have been identified which are found to be responsible for these conditions.
Deven Dhurandhar, Niroj Kumar Sahoo, Indumathi Mariappan, Raja Narayanan   +3 more
doaj   +1 more source

First results of long-term follow-up of children in Russia after gene therapy for hereditary retinal dystrophies associated with biallelic mutations in the RPE65 gene

Российский офтальмологический журнал, 2023
Purpose: to evaluate the results of gene therapy by the recombinant adeno-associated viral vector voretigene neparvovec (VN) in children with follow-up periods of 1, 3, 6, and 12 months.Material and methods. The study included 6 children (12 eyes) aged 5
V. V. Neroev, L. A. Katargina, M. P. Kharlampidi, L. V. Kogoleva, I. V. Zolnikova, P. A. Ilyukhin, E. V. Denisova, S. V. Milash, N. A. Osipova, S. I. Kutsev, A. V. Polyakov, R. A. Zinchenko, V. V. Kadyshev, Yu. A. Bobrovskaya   +13 more
doaj   +1 more source

The Multiple Criteria Qualitative Value‐Based Pricing Framework “MARIE” for Novel Cell and Gene Therapy

Clinical Pharmacology &Therapeutics, EarlyView.
When setting the price of cell and gene therapies in Japan, the factors considered include the manufacturing and operating costs of the new drug or a comparator, leading to discrepancies between price and values, in particular those specific to cell and gene therapies.
Akina Takami, Ataru Igarashi
wiley   +1 more source

Clinical manifestations of dual‐gene variants in retinitis pigmentosa

Acta Ophthalmologica, EarlyView.
Abstract Purpose Retinitis pigmentosa (RP) is an inherited retinal disease (IRD), whereby each affected individual typically harbours pathogenic variants in a single causative gene, yet the disorder exhibits marked genetic heterogeneity, with more than 100 genes reported to underlie RP.
Lasse Wolfram, Jan‐Philipp Bodenbender, David A. Merle, Milda Reith, Anton Sonntag, Krunoslav Stingl, Tobias B. Haack, Theresia Zuleger, Bernd Wissinger, Laura Kühlewein, Katarina Stingl, Susanne Kohl   +11 more
wiley   +1 more source

Techniques for subretinal injections in animals

Veterinary Ophthalmology, Volume 28, Issue 2, Page 506-518, March 2025.
Abstract Subretinal injections are not commonly performed during clinical treatment of animals but are frequently used in laboratory animal models to assess therapeutic efficacy and safety of gene and cell therapy products. Veterinary ophthalmologists are often employed to perform the injections in the laboratory animal setting, due to knowledge of ...
Ryan F. Boyd, Simon M. Petersen‐Jones
wiley   +1 more source

Subretinal Injection of Voretigene Neparvovec-rzyl in a Patient With RPE65-Associated Leber's Congenital Amaurosis

, 2019
Leber's congenital amaurosis (LCA) is a rare inherited retinal degeneration (IRD) that causes severe vision loss, nyctalopia, and nystagmus within the first few years of life.
Hussain, Rehan M, Berrocal, Audina M, Tran, Kimberly D, Maguire, Albert M   +3 more
core   +1 more source