References in periodicals archive ?
In January 2017, Acucela also received orphan drug designation for emixustat for the treatment of Stargardt disease by FDA (US Food and Drug Administration).
LBS-008 received US and EU orphan drug designation (ODD) in 2017 and 2018 and rare paediatric disease designation (RPD) from the US FDA in 2018 for the treatment of Stargardt disease.
Singh et al., "Mutation of the Stargardt disease gene (ABCR) in age-related macular degeneration," Science, vol.
This report provides comprehensive information on the therapeutic development for Juvenile Macular Degeneration (Stargardt Disease), complete with comparative analysis at various stages, therapeutics assessment by drug target, mechanism of action (MoA), route of administration (RoA) and molecule type, along with latest updates, and featured news and press releases.
Both age-related macular degeneration and Stargardt disease are currently deemed untreatable.
"This is important because stem cell derived RPE are currently in clinical trials for the treatment of age related macular degeneration and Stargardt disease," said Anthony Atala, MD, Editor of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine.
Genetic eye disease (GED) is one of the leading causes of blindness and includes disorders affecting all structures of the eye from the anterior to posterior segment, such as albinism, corneal dystrophy, aniridia, primary glaucoma, pediatric cataracts, retinitis pigmentosa, Stargardt disease, and hereditary optic neuropathy.
To be here and to win bronze is fantastic." Ben took up judo when he was seven but was diagnosed with Stargardt disease - a rare genetic condition - three years later.
Ashlea - mum to Jessica, eight, Brooke, five, Grace-May, three, and newborn Esme - was the first of the Manchester sisters to be diagnosed with Stargardt disease. She was expecting Grace-May at the time.
Doctors at Moorfields Eye Hospital in London will transplant laboratory-grown retinal cells into patients with advanced Stargardt disease.
Doctors at Moorfields Eye Hospital in London will transplant laboratory-grown retinal cells into patients with advanced Stargardt disease. The Phase I study, which should take place within the next few months, is designed to investigate the safety of the treatment.
"There is real potential that people with disorders including Stargardt disease and age-related macular degeneration (AMD)might benefit," the Daily Express quoted Professor James Bainbridge, who will be conducting the trials, as saying.