Opus Genetics has completed a Type B Regenerative Medicine Advanced Therapy (RMAT) meeting with the US Food and Drug Administration (FDA) regarding OPGx-LCA5, its gene therapy candidate for Leber ...
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 ...
Relationship Among DNA Damage Response Gene Alterations, Molecular Subtypes, and Survival Outcomes in Patients With Metastatic Bladder Cancer Treated on CALGB 90601 Sequencing data from 56,965 ...
A newly identified mutation helps super-sleepers get by on just four to six hours of shut-eye per night, while the rest of us need around eight hours. Researchers described the SIK3-N783Y mutation in ...
Outcomes of Radium-223 and Stereotactic Ablative Radiotherapy Versus Stereotactic Ablative Radiotherapy for Oligometastatic Prostate Cancers: The RAVENS Phase II Randomized Trial We analyzed a cohort ...
A groundbreaking discovery from Israeli scientists is changing what doctors know about inherited heart disease. Researchers have pinpointed a gene, TRIM63, as a major contributor to hypertrophic ...
Not revised: This Reviewed Preprint includes the authors’ original preprint (without revision), an eLife assessment, and public reviews. Even though this is not the first report that the mutation in ...
First patient dosed in the pediatric cohort of the Phase 1/2 trial of OPGx-LCA5; initial data on the cohort anticipated by Q3 2025 New 12-month data on the first three adult OPGx-LCA5 patients to be ...
Stargardt disease, the most common form of juvenile macular degeneration, results from biallelic mutations in the ABCA4 gene. In a recently published study, researchers at the Institute of Molecular ...
Atsena Therapeutics and Nippon Shinyaku have formed an exclusive licensing agreement for ATSN-101, a gene therapy for Leber congenital amaurosis (LCA1). Nippon Shinyaku will commercialize ATSN-101 in ...
Background: Oligoasthenoteratozoospermia (OAT) is a widespread cause of male infertility. One of the usual clinical manifestations of OAT is multiple morphological abnormalities of the sperm flagella ...
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