- The European Medicines Agency granted orphan designation to US-based ophthalmology company Acucela Inc's leading drug candidate emixustat hydrochloride for the treatment of Stargardt disease
, the company said.
According to the company, it holds the worldwide exclusive rights for LBS-008, a first-in-class oral therapy that prevents the buildup of toxins (A2E) in the eye that cause Stargardt disease
and contribute to atrophic Age-related Macular Degeneration (dry AMD).
Regarding phenotype and severity of visual symptoms, the family showed Stargardt disease
at various stages of progression.
Test-Retest Variability of Functional and Structural Parameters in Patients with Stargardt Disease
Participating in the SAR422459 Gene Therapy Trial.
"To date, it has been difficult to obtain great quantities of human cones." His discovery offers a way to overcome this problem, offering hope that treatments may be developed for currently non-curable degenerative diseases, like Stargardt disease
[USPRwire, Wed Sep 09 2015] Global Markets Direct's, 'Juvenile Macular Degeneration (Stargardt Disease
) - Pipeline Review, H2 2015', provides an overview of the Juvenile Macular Degeneration (Stargardt Disease
)'s therapeutic pipeline.
(2015) Early-onset stargardt disease
: Phenotypics and genotypic characteristics.
Omeasoo, who has Stargardt disease
and is legally blind, did not notice the camera crews that had been set up in the bank.
The discovery could also help children with Stargardt disease
, the main cause of blindness in the young.
Ychydig cyn ei phen-blwydd yn 10 oed cafodd wybod bod ganddi gyflwr o'r enw 'Stargardt Disease
', sef math o nam golwg.
(STGD) is a macular degeneration that occurs in childhood, resulting in a profound loss of central vision and severely reduced visual acuity.
She was diagnosed with Stargardt disease
, which affects one in 10,000 young people.