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Gene Therapy Shows Robust Activity for Duchenne Muscular DystrophyA novel gene therapy has demonstrated impressive early results in a small sample of 3 children with Duchenne muscular dystrophy.
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Rare Neurologic Diseases Are Not Truly RareDespite being labeled as rare diseases, a number of neurologic conditions impact more patients than most would believe. The consultant with expertise in ophthalmology, gene therapy, and rare and orphan diseases, chimed in about how these diseases can often be overlooked.
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Ian Miller, MD: Adapting AAV Gene Therapy for SCN1A-Positive EpilepsyThe medical director of the Comprehensive Epilepsy Clinic at Nicklaus Children’s Hospital discusses his early stage gene therapy trial for Dravet syndrome.
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Parkinson and Lewy Body-like Dementia TriggersThe discovery of a possible trigger for PD and Lewy body dementia could provide a new avenue for gene therapy in these neurological conditions.
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CGRPs: New Help in Migraine Prophylaxis?A new class of injectable monoclonal antibodies directed against calcitonin gene related peptide (CGRP) or its receptor may offer hope to migraineurs who have not found effective prophylactic therapy.
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Gene Therapy Ushers in an Era of Hope for Rare DisordersPreclinical trials and success stories suggest that much is riding on vector-based therapies for the treatment of rare neurological conditions.
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Dystrophin Pathway and Treatments Offer Hope for Patients with Duchenne Muscular DystrophyGene therapy has generated excitement as a treatment or even a potential cure for inherited diseases. Among them: Duchenne muscular dystrophy.
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Large Gene Network Required to Predict Response to HER2-Targeted TherapyLajos Pusztai, MD, DPhil, discusses findings that suggest it is unlikely that any single gene can predict response to targeted therapy for patients with HER2-positive breast cancer.
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Steven Buechler Discusses EarlyR Gene Signature in the BIG 1-98 Trial for Breast CancerSteven Buechler, PhD, Department of Applied and Computational Mathematics and Statistics at the University of Notre Dame, discusses independent validation of the EarlyR gene signature in the Breast International Group (BIG) 1-98 study. This randomized, double-blind, phase III trial compared letrozole with tamoxifen as an adjuvant endocrine therapy for postmenopausal women with hormone receptor-positive, early breast cancer
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Sintilimab/Pemetrexed Regimen Significantly Improves PFS in Advanced NSCLCSintilimab injection plus pemetrexed and platinum-based therapy led to a statistically significant improvement in progression-free survival compared with chemotherapy alone as a first-line treatment for patients with locally advanced or metastatic nonsquamous non–small cell lung cancer.
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What's Keeping CAR T-Cell Therapies From the Outpatient Setting?Although these therapies were initially conceived of and developed as inpatient therapies, interest is growing in extending chimeric antigen receptor T-cell therapies to the outpatient setting.
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Biomarkers and Histology Play Key Roles in Frontline Metastatic NSCLC OptionsIn light of recent advancements, the current paradigm for choosing first-line therapy for patients with metastatic non–small cell lung cancer who do not harbor an actionable driver oncogene depends upon PD-L1 expression level and histology.
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News Reports; March 2007News items reported in this issue: 1) 186-Gene Signature in Cancer Stem Cells Predicts Recurrence 2) National Prostate Cancer Coalition Commences Clinical Trial Education Program 3) Concomitant High-Dose Radiation Therapy Plus Cetuximab Improves Locoregional Control and Reduces Mortality, with No Increase in Radiation Therapy%u2013Associated Toxicity in Patients with Advanced Squamous-Cell Carcinoma of the Head and Neck, and more
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Updated CRYSTAL Data Support Effectiveness of Cetuximab (Erbitux) in Wild-Type KRAS Metastatic Colorectal CancerAt the 2010 Gastrointestinal Cancers Symposium, new data from the CRYSTAL trial identified BRAF gene mutations as a poor prognostic indicator in metastatic colorectal cancer (mCRC) but not predictive of response to therapy.
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Working Toward a Cure in Follicular LymphomaA panel of lymphoma experts discussed 2 recently approved treatments that are providing hope to patients with relapsed or refractory follicular lymphoma.
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New Therapies for Age-Related Macular DegenerationChristina Y. Weng, MD, MBA, Baylor College of Medicine, Houston, discussed therapies for macular degeneration.
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New Study Explores Neurocognitive Outcomes Following CAR T-Cell TherapyAs patients who undergo CAR T-cell therapy survive longer, more research is required into the long-term neurocognitive effects of this treatment.
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JAMA Commentary Urges Realistic View of New CAR T-Cell Cancer TherapyWhile the FDA’s approval of the immunotherapy tisagenlecleucel represents a landmark in the oncology field, the treatment is far from being a cure and should be viewed in context of its $475,000 price tag, a group of oncologists wrote in a commentary published in JAMA.
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Supporting Translational Cell Therapy ResearchRenier Brentjens, MD, PhD, the chair of the department of medicine at Roswell Park Comprehensive Cancer Center, discussed the need to bring more innovations from nonclinical research to patients.
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Back to the Bench: Exploring the Potential of CAR T-Cell Therapy in OncologyMiguel-Angel Perales, MD, and Caron A. Jacobson, MD, share insights regarding the context and implications of the recent actions by the FDA to add boxed warnings to CAR T-cell therapies.
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Dr. Perl on CAR T Cells in Pediatric LeukemiaAlexander E. Perl, MD, associate professor of medicine, University of Pennsylvania, Perelman School of Medicine, discusses the promise of CAR T cells in the treatment of pediatric leukemia.
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Dr. Younes Discusses the Future of CAR T-Cell TherapyAnas Younes, MD, chief of Lymphoma Service, Memorial Sloan Kettering Cancer Center, discusses the future of chimeric antigen receptor (CAR) T-cell therapy for patients with hematologic malignancies.
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Dr. Htut on CAR T-Cell Therapy in Patients With Multiple MyelomaMaung Myo Htut, MD, assistant clinical professor of hematology and hematopoietic cell transplantation, City of Hope, discusses the use of chimeric antigen receptor (CAR) T-cell therapy in patients with multiple myeloma.
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Dr. Sasine on CAR T-Cell Therapy in Ovarian CancerJoshua P. Sasine, MD, medical director, Chimeric Antigen Receptor T-Cell Program, David Geffen School of Medicine, University of California, Los Angeles, discusses chimeric antigen receptor (CAR) T- cell therapy in ovarian cancer.
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Brexucabtagene Autoleucel in R/R Mantle Cell LymphomaExperts discussed the ZUMA-2 clinical trial of brexucabtagene autoleucel.
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Lead Investigator Shares Early Data With Ibrutinib Maintenance in MCLReem Karmali, MD, MS, shares early data with ibrutinib maintenance therapy and highlights recent advances and challenges in the treatment of patients with mantle cell lymphoma.
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CARTITUDE Trials in Multiple MyelomaExperts discussed safety and efficacy data of cilta-cel for R/R MM from the CARTITUDE-1 and CARTITUDE-2 studies.
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Dr. Ostertag on Emerging CAR T Product for MyelomaEric M. Ostertag, MD, PhD, chief executive officer, Poseida Therapeutics, Inc., discusses an emerging CAR T stem cell memory product for patients with relapsed/refractory multiple myeloma.
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Monitoring and Support for Patients Receiving CAR T-Cell TherapyExperts discuss providing supportive care to patients receiving CAR T-cell therapy.
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Dr. O'Brien on CAR T Cells in CLLSusan M. O’Brien, MD, hematologist/oncologist at University of California, Irvine Health, discusses the potential for CAR T-cell therapy for the treatment of chronic lymphocytic leukemia.
