More early-stage data show the drug, called salanersen, appeared to have positive effects on neurodegeneration and motor function in patients previously treated with Novartis' Zolgensma.

Original title: “Biogen builds case for Spinraza successor with fresh data”

Ophthalmology Times connects eye care professionals with surgery, imaging, gene therapy, & diagnostic advances to enhance clinical and patient care. In recent years, gene therapy has emerged from the shadows of theoretical potential into the spotlight of clinical reality. The approval of voretigene neparvovec-rzyl (Luxturna; Spark Therapeutics) marked a historic moment as the first gene therapy for an inherited retinal disease (IRD) approved in Europe and the US. Operational challenges in gene therapy include specialized infrastructure, multidisciplinary teams, and patient education for realistic expectations and adherence. Research is expanding to ABCA4-related diseases using novel vector technologies, addressing delivery limitations and enabling larger transgene payloads. Gene therapy is not a cure but rather a functional intervention, and setting realistic goals is essential for long-term satisfaction and follow-up adherence. An area of intense focus at our center is ABCA4-associated retinal degeneration such as Stargardt disease, the most common form of juvenile hereditary retinopathy. We are now preparing to initiate a phase 1 clinical trial investigating gene therapy for ABCA4-related disease, supported by a novel adeno-associated virus (AAV)–based dual vector technology developed by VeonGen Therapeutics (formerly known as ViGeneron). This platform was codeveloped by Stylianos Michalakis, professor at the University Eye Hospital of LMU Munich and one of the company’s cofounders.

Original title: “From bench to bedside: Evolving realities of gene therapy for inherited retinal diseases | Ophthalmology Times - Clinical Insights for Eye Specialists”

ormdl proteins turnover via proteasome and autophagy is cell type dependent and tied to ceramide homeostasis

Original title: “ORMDL Proteins Turnover via Proteasome and Autophagy Is Cell-Type Dependent and Tied to Ceramide Homeostasis.”

glucokinase inactivation reduces lysophosphatidylcholine accumulation to alleviate high fat diet induced hepatic steatosis

Original title: “Glucokinase Inactivation Reduces Lysophosphatidylcholine Accumulation to Alleviate High-Fat Diet-Induced Hepatic Steatosis.”

delpacibart etedesiran improves the molecular pathology of myotonic dystrophy type 1 in the phase 1 2 marina study

Original title: “Delpacibart Etedesiran Improves the Molecular Pathology of Myotonic Dystrophy Type 1 in the Phase 1/2 MARINA® Study.”

missense variants in the a isoform of fgf13 as a novel cause of paroxysmal dyskinesia

Original title: “Missense Variants in the A Isoform of FGF13 as a Novel Cause of Paroxysmal Dyskinesia.”

sort lnps encapsulating cas9 mrna achieve efficient editing in skeletal muscle in a dystrophic mouse model

Original title: “SORT LNPs encapsulating Cas9 mRNA achieve efficient editing in skeletal muscle in a dystrophic mouse model.”

evaluation of mixed response in tumor size and survival in patients with rare cancers treated with dual checkpoint inhibitor therapy dart swog s1609

Original title: “Evaluation of mixed response in tumor size and survival in patients with rare cancers treated with dual checkpoint inhibitor therapy (DART SWOG S1609).”

long term follow up of pleuroparenchymal fibroelastosis associated with crohn s disease

Original title: “Long-Term Follow-Up of Pleuroparenchymal Fibroelastosis Associated With Crohn's Disease.”

clinical characteristics and anti zscan1 antibody titer analysis in a nationwide survey of rohhad net syndrome

Original title: “Clinical characteristics and anti-ZSCAN1 antibody titer analysis in a nationwide survey of ROHHAD (-NET) syndrome.”

outcomes of sars cov 2 infection post solid organ transplantation in the cystic fibrosis population

Original title: “Outcomes of SARS-CoV-2 infection post-solid organ transplantation in the cystic fibrosis population.”

renal schwannoma appears on imaging as renal cell carcinoma a case report

Original title: “Renal schwannoma appears on imaging as renal cell carcinoma: a case report.”

guideline based diagnosis and management of congenital platelet function disorders current insights and future directions

Original title: “Guideline-based diagnosis and management of congenital platelet function disorders: Current insights and future directions.”

extranodal rosai dorfman disease of the male breast a case report and literature review

Original title: “Extranodal Rosai-Dorfman Disease of the Male Breast: A Case Report and Literature Review.”

regional variations and trends in hemophilia prevalence a global analysis with future projection

Original title: “Regional variations and trends in hemophilia prevalence: A global analysis with future projection.”

advanced cardiac imaging in cardiac sarcoidosis current evidence and future directions

Original title: “Advanced cardiac imaging in cardiac sarcoidosis: current evidence and future directions.”

optimising primary therapy in primary biliary cholangitis opera protocol for a randomised double blind placebo controlled trial of enhanced primary therapy with obeticholic acid

Original title: “Optimising Primary thErapy in pRimAry biliary cholangitis (OPERA): protocol for a randomised, double-blind, placebo-controlled trial of enhanced primary therapy with obeticholic acid.”

old and emerging therapies for childhood interstitial lung disease child a systematic review

Original title: “Old and emerging therapies for childhood interstitial lung disease (chILD): a systematic review.”

original research amplification of genetic and metabolic factors in alpha 1 antitrypsin deficiency

Original title: “Original research: Amplification of genetic and metabolic factors in alpha-1 antitrypsin deficiency.”