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The director of the Sickle Cell Disease Program at Nemours Children’s Hospital discussed what clinics need in order to start administering gene therapy effectively. Some of them have already failed disease modifying therapies and are really looking for something else, but some haven't tried disease modifying therapies yet, and we might direct them that way first.” · Vertex Pharmaceuticals' and CRISPR Therapeutics’ gene editing therapy · exagamglogene autotemcel (exa-cel; marketed as Casgevy) and bluebird bio’s gene addition therapy · lovotibeglogene autotemcel (lovo-cel; marketed as Lyfgenia) were approved by the FDA for the treatment of sickle cell disease (SCD) in late 2023. 67th American Society of Hematology (ASH) Annual Meeting and Exposition, held December 6 to 9, 2025, in Orlando, Florida, CGTLive® spoke with Robin E. Miller, MD, the director of the Sickle Cell Disease Program at Nemours Children’s Hospital, to learn more about the practical considerations for treatment centers that are integrating or considering integrating gene therapy into their clinical care for SCD. Miller stressed that centers need key infrastructure, including SCD specialists, transplant experts, a robust blood bank, a stem cell lab, and experience with bone marrow transplant in SCD. Also important are psychosocial supports, including psychologists and social workers familiar with the unique challenges that families of patients with SCD face. She also explained that determining candidacy requires multidisciplinary input and shared decision making. Families must understand that while gene therapy is promising, it is a long process and carries risks. Some patients may be better suited for other therapies or traditional stem cell transplant, especially if a matched donor is available. Miller emphasized the importance of clearly explaining risks, benefits, and practical factors like recovery time and long-term follow-up. ... Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.

Original title: “Robin E. Miller, MD, on Integrating Gene Therapy into Clinical Care for Sickle Cell Disease | CGTlive®”

incidence and characteristics of hbv reactivation related events following hbsag seroclearance long term follow up of a population based cohort in japan

Original title: “Incidence and Characteristics of HBV Reactivation-Related Events Following HBsAg Seroclearance: Long-Term Follow-up of a Population-Based Cohort in Japan.”

phase ii clinical trial of kcho 1 for alzheimer s disease study protocol for a multicenter randomized double blind multi dose placebo trial

Original title: “Phase II clinical trial of KCHO-1 for Alzheimer's disease: study protocol for a multicenter, randomized, double-blind, multi-dose, placebo trial.”

incidence and severe outcomes of acute kidney injury in haff disease a brazilian cohort study

Original title: “Incidence and Severe Outcomes of Acute Kidney Injury in Haff Disease: A Brazilian Cohort Study.”

identification and bioinformatics analysis of malate dehydrogenase and pyruvate kinase as novel allergens in cicada pupae

Original title: “Identification and Bioinformatics Analysis of Malate Dehydrogenase and Pyruvate Kinase as Novel Allergens in Cicada Pupae.”

novel translational pulmonary mri in pediatrics a review of the last 10 years

Original title: “Novel translational pulmonary MRI in pediatrics: A Review of the last 10 years.”

dose finding and optimization in drug development for rare diseases

Original title: “Dose-finding and optimization in drug development for rare diseases.”

facial nerve outcomes following vestibular schwannoma surgery multivariate analysis of possible prognostic factors

Original title: “Facial nerve outcomes following vestibular schwannoma surgery: Multivariate analysis of possible prognostic factors.”

carpal tunnel syndrome caused by parosteal lipoma of carpal bones a case report

Original title: “Carpal Tunnel Syndrome Caused by Parosteal Lipoma of Carpal Bones: A Case Report.”

tuberculosis associated nonbacterial thrombotic endocarditis a case report

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case of eosinophilic granulomatosis with polyangiitis emerging during benralizumab therapy successful management through a switch to mepolizumab therapy

Original title: “A Case of Eosinophilic Granulomatosis with Polyangiitis Emerging During Benralizumab Therapy: Successful Management Through a Switch to Mepolizumab Therapy.”