M2 PHARMA-June 11, 2019-Acucela Receives Orphan Designation from the EMA for Emixustat for the Treatment of Stargardt
M2 EQUITYBITES-April 25, 2019-Belite Bio wins US FDA approval for LBS-008 IND for macular degeneration and Stargardt
Global Banking News-April 25, 2019-Belite Bio wins US FDA approval for LBS-008 IND for macular degeneration and Stargardt
Italian journalist Paola Peretti discovered as a young girl that she was suffering progressive loss of vision caused by the incurable Stargardt
Wave's research in ophthalmology will initially focus on the following four inherited retinal diseases which commonly lead to progressive vision loss typically starting in childhood or adolescence: retinitis pigmentosa due to a P23H mutation in the RHO gene, Stargardt
disease, Usher syndrome type 2A and Leber congenital amaurosis 10.
's disease, also known as Stargardt
's macular dystrophy, affects the area of the retina called the macula and causes a reduction in central vision.
"There is no approved treatment available for Stargardt
's, so we are very excited about the opportunity to test remofuscin and offer these patients the hope that we may be able to stop the progression of the condition." Professor Carel Hoyng, specialist in inherited retinal diseases at Radboud University Medical Centre in the Netherlands, said: "This drug's potential goes beyond existing approaches.
Mutations in ABCA4 gene are known to cause Stargardt
disease (STGD) which is the most frequent macular dystrophy  and typically presents with central macular atrophy and yellow-white dots at the posterior pole, primarily at the level of the RPE.
Fourteen-year-old Abby, who suffers from Stargardt
disease, a condition that leads to progressive vision deterioration, previously finished sixth in the Paralympics.
Will Kent describes his vision as "like looking in to space." Will and his mother, Laura Kent, talk with OT about Stargardt
disease and their hopes for the future
By Nicholas Stargardt
. (New York, NY: Basic Books, 2015.
Attendees observed a student recital featuring WMU horn players and a master class including solo competition winner Eden Stargardt
. Lin and Leelanee performed solos in the final concert, after which college students and participants alike took the stage and engulfed the hall in the sound of 45 horns.