There is preclinical evidence that ataluren has a therapeutic effect in animal and cell models of aniridia, choroideremia
, Usher syndrome, and retinitis pigmentosa caused by RP2, MERTK and CEP290.
- The US Food and Drug Administration has granted orphan drug designation to 4D-110 for the treatment of choroideremia
, US-based gene therapeutic products developer 4D Molecular Therapeutics said.
(114.) EudraCT number 2015-001383-18 Open open Label Phase 2 Clinical Trial of Retinal Gene Therapy for Choroideremia
Using an Adeno-Associated Viral Vector (AAV2) Encoding Rab-Escort Protein 1 (REP1).
Hot on the heels of the AAV-RPE65 trial, phase 1/2 clinical trials for the X-linked chorioretinopathy choroideremia
were initiated [36, 37] following preclinical studies in [Chm.sup.null/WT] mice .
Williams, in partnership with Athletes Brand, co-designed a t-shirt in Yankees-themed colors that is being sold to raise proceeds and awareness for the Choroideremia
Spark Therapeutics, a United States-based gene therapy company, has commenced enrolment in a Phase I/II clinical trial of its product candidate, SPK-CHM to treat patients with choroideremia
, an X-linked inherited retinal dystrophy, it was reported yesterday.
He was a member of a Tuesday night bowling league and was a member of the Choroideremia
Jonathan Wyatt, 65, and Toby Stroh, 56, were born with a genetic condition called choroideremia
which causes people to slowly lose their sight.
is an X-linked disorder, which means that it is caused by a faulty gene, called CHM, on the X chromosome.
looked at six people, diagnosed with choroideremia
is a rare condition that occurs in different stages, starting with night blindness in early childhood, gradually ending up with permanent blindness by middle age.