Addition of TUS to VEN+AZA achieves CR/CRh responses in all (6/6, 100%) patients treated at the higher dose levels of 80 mg and 120 mg TUS, ...

Neuronal ceroid lipofuscinosis type 1 (CLN1) disease, also known as infantile neuronal ceroid lipofuscinosis, is a rare and fatal neurodegenerative condition. CLN1 disease is caused by a deficiency in ...

AAVB-039 addresses the root cause of the disease, benefitting patients with any ABCA4 mutation AAVB-039 is currently being assessed in the CELESTE interventional clinical trial and we continue ...

the company’s investigational gene therapy for the treatment of Stargardt disease secondary to biallelic mutation in ABCA4, Orphan Drug Designation (ODD). The company has also received Clinical Trial ...

Opus Genetics today announced the positive 3-month pediatric data from its Phase 1/2 open-label, ascending-dose study of the safety and efficacy of OPGx-LCA5, an investigational gene therapy for Leber ...

Pediatric participants demonstrated large gains in cone-mediated vision; therapy remains well tolerated with no ocular serious adverse events or dose-limiting toxicitiesLasting, durable responses ...

Cancers are caused by mutations in driver genes. The impact of those mutations in the cell can be influenced by other proteins in the cell that physically interact with the mutated protein. In this ...

The FDA has awarded orphan drug designation to Cure Rare Disease’s CRD-003 for the treatment of limb-girdle muscular dystrophy type R9 (LGMD2i/R9), a congenital muscular dystrophy caused by biallelic ...

The real money (and fun) in Roblox Build a Zoo starts when you unlock Big Pets and start experimenting with mutations. Since these two systems work so well hand in hand, I decided to mix them in a ...

Decades ago, India’s tigers were on the brink of extinction. Slowly, their numbers have rebounded. But that ecological success has prompted a dire problem—and a race to save many of them from genetic ...