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  • ||||||||||  Adstiladrin (nadofaragene firadenovec-vncg) / Ferring
    Enrollment change:  ABLE-41: ADSTILADRIN Early Utilization and Outcomes in the Real World Setting (clinicaltrials.gov) -  Nov 13, 2024   
    P=N/A,  N=400, Recruiting, 
    This GWAS, the largest pharmacogenomic study of response to NTZ, suggested MS-implicated genes and Wnt/?-catenin signaling pathway, an essential component for blood-brain barrier formation and maintenance, to be related to treatment response. N=800 --> 400
  • ||||||||||  Association between Targeted Therapy and Survival in Patients with MDS (Halls G-H (San Diego Convention Center)) -  Nov 6, 2024 - Abstract #ASH2024ASH_5741;    
    In addition, our study confirms that a significant number of biomarker-positive MDS patients do not receive targeted therapies, highlighting challenges such as disparities in access to care and provider awareness. Addressing these issues is essential to ensure all eligible patients benefit from advancements in targeted therapies, ultimately improving survival and quality of life.
  • ||||||||||  Venclexta (venetoclax) / Roche, AbbVie, Idhifa (enasidenib) / BMS, Servier, Tibsovo (ivosidenib) / Servier
    Single-Cell Proteogenomic Analysis of Clonal Evolution in Patient-Derived Xenograft Models of AML Treated with IDH Inhibitors (Halls G-H (San Diego Convention Center)) -  Nov 6, 2024 - Abstract #ASH2024ASH_4322;    
    Here, we used single-cell DNA sequencing (scDNA-seq) and surface protein profiling to monitor the clonal evolution and differentiation of an isocitrate dehydrogenase 1 (IDH1)-mutated leukemic sample in response to ivosidenib (IVO), a mutant IDH1 inhibitor, either as monotherapy or in combination with venetoclax (VEN) or azacitidine (AZA)...As proof-of-principle, we generated an IDH1R132H/IDH2R140Q mPDX to model isoform switching in both directions by treating the animals with either IVO or enasidenib (ENA), a mutant IDH2 inhibitor...Further studies are underway to demonstrate whether dual mutant IDH1 and IDH2 inhibition (IVO+ENA) will circumvent resistance driven by isoform switching. In summary, we demonstrate the utility of applying single-cell proteogenomic analysis in traditional and mixed PDX models to gain crucial insights into mechanisms of resistance and potential strategies to overcome it.
  • ||||||||||  Venclexta (venetoclax) / Roche, AbbVie
    Venetoclax Enhances Human ??t Cells Anti-Leukemia Immunity through Metabolic Reprogramming (Halls G-H (San Diego Convention Center)) -  Nov 6, 2024 - Abstract #ASH2024ASH_3757;    
    Combining Venetoclax with ??T cells decreased tumor burden in THP-1-bearing mice. We found upregulated activation markers CD25 and CD69 and cytotoxic molecules NKG2D and DNAM-1 on drug-treated ??T cells when these cells were exposed to tumor targets.
  • ||||||||||  Idhifa (enasidenib) / BMS, Servier, Onureg (azacitidine oral) / BMS
    Opening Pandora's Box: Ultra-Deep Duplex Sequencing in Long Survivors with Acute Myeloid Leukemia (Halls G-H (San Diego Convention Center)) -  Nov 6, 2024 - Abstract #ASH2024ASH_2953;    
    However, low-level persistence and/or appearance of mutations in known pathogenic hotspots were frequently observed after years of ongoing remission, including for pts receiving maintenance. Further investigation of the clinical relevance of low-level mutations in long-term remission is needed to inform decision-making and prevent over-treatment.
  • ||||||||||  Association between Targeted Therapy and Survival in Patients with AML (Halls G-H (San Diego Convention Center)) -  Nov 6, 2024 - Abstract #ASH2024ASH_2854;    
    However, a significant number of biomarker-positive AML patients do not receive targeted therapies, highlighting challenges such as disparities in access to care and provider awareness. Addressing these issues is essential to ensure all eligible patients benefit from advancements in targeted therapies, ultimately improving survival and quality of life.
  • ||||||||||  Spinraza (nusinersen) / Biogen
    Study on the Economic Burden and Multi-Level Medical Security System of SMA Patients in Shaanxi Province, China () -  Nov 4, 2024 - Abstract #ISPOREU2024ISPOR_EU_2836;    
    While the multi-tiered medical security system has somewhat reduced the financial burden on SMA patients, families still face high medical expenses due to low incomes and insufficient labor force. It is recommended to further enhance policy interventions, improve the accessibility and affordability of rare disease drugs, and optimize the medical security system.
  • ||||||||||  Zolgensma (onasemnogene abeparvovec-xioi) / Novartis, Evrysdi (risdiplam) / SMA Foundation, PTC Therap, Roche, Spinraza (nusinersen) / Biogen
    Healthcare Pathways and Therapeutic Outcomes of Patients With Spinal Muscular Atrophy: Results From the 12-Year Real-World Study EPI-SMA Based on the French National Healthcare Database (SNDS) () -  Nov 4, 2024 - Abstract #ISPOREU2024ISPOR_EU_2529;    
    OBJECTIVES: Considering the growing number of prescriptions of innovative therapies for Spinal Muscular Atrophy (SMA) (nusinersen [2017], onasemnogene abeparvovec [2019] and risdiplam [2020]) in France, the EPI-SMA study aimed to provide real-world data on the epidemiology, management of therapeutic options, patient journeys, costs and impact on overall survival from 2011 up to 2022... This real-world study confirms that SMA therapies have significantly impacted the healthcare pathways of SMA patients, particularly those with the most severe patients (SMA1-like), and have contributed to increased overall survival.
  • ||||||||||  Zolgensma (onasemnogene abeparvovec-xioi) / Novartis, Evrysdi (risdiplam) / SMA Foundation, PTC Therap, Roche, Spinraza (nusinersen) / Biogen
    Consolidating Methods of Cost-Utility Analysis for Newborn Screening of Spinal Muscular Atrophy: A Systematic Review () -  Nov 4, 2024 - Abstract #ISPOREU2024ISPOR_EU_2505;    
    Two studies included three treatments (Nusinersen, Onasemnogene abeparvovec, Risdiplam), two included two treatments (Nuisnersen, Onasemnogene aveparvovec) and one included only nusinersen... The findings from this study can provide reliable data inputs for future cost-utility analysis studies evaluating newborn screening for SMA.
  • ||||||||||  Zolgensma (onasemnogene abeparvovec-xioi) / Novartis, Evrysdi (risdiplam) / SMA Foundation, PTC Therap, Roche, Spinraza (nusinersen) / Biogen
    Implementing Health Technology Management of Medicines for Rare Diseases: A Case Study of Reimbursement of Medicines for Spinal Muscular Atrophy in Ireland () -  Nov 4, 2024 - Abstract #ISPOREU2024ISPOR_EU_2383;    
    We review the eligibility criteria applied for medicines for SMA (nusinersen, onasemnogene abeparvovec [OA], risdiplam) under the MAP approach in Ireland and provide an overview of applications received to date... MAPs have been used to facilitate access to medicines for SMA in Ireland, while providing the healthcare payer with certainty that treatments will only be reimbursed in the approved cohorts aligned with those considered in the formal pricing and reimbursement process.
  • ||||||||||  Trelegy Ellipta (fluticasone furoate/umeclidinium bromide/vilanterol) / GSK, Innoviva, Theravance Biopharma
    Decomposing Overall Mortality Into Latent Disease-Specific Health States Mortality to Inform Cost-Effectiveness Modeling () -  Nov 4, 2024 - Abstract #ISPOREU2024ISPOR_EU_2293;    
    The methodology to decompose overall mortality into health state-specific mortality, accounting for general population mortality, was successfully implemented. This approach can be applied in health economic models to predict health state specific mortality from overall mortality accounting for general population mortality.
  • ||||||||||  Spinraza (nusinersen) / Biogen
    Journal:  Dysregulated balance of D- and L-amino acids modulating glutamatergic neurotransmission in severe spinal muscular atrophy. (Pubmed Central) -  Nov 1, 2024   
    To address this issue, we measured the levels of the main neuroactive D- and L-amino acids acting on glutamatergic receptors in the central nervous system of SMN?7 mice as well as the cerebrospinal fluid (CSF) of SMA patients of varying severity before and after treatment with the SMN-inducing drug Nusinersen...Lastly, consistent with a correlation of higher CSF levels of D-serine with better motor function in severe SMA patients, we show that daily supplementation with the NMDA receptor co-agonist D-serine improves neurological deficits in SMN?7 mice. Altogether, these findings provide direct evidence for dysregulation of D- and L-amino acid metabolism linked to glutamatergic neurotransmission in severe SMA and have potential implications for treating this neurological disorder.