Results 121 to 130 of about 13,301,732 (374)
Innovative Insurance to Improve US Patient Access to Cell and Gene Therapy
The Milbank Quarterly, Volume 103, Issue 1, Page 32-51, March 2025.Policy Points Cell and gene therapies (CGTs) offer treatment for rare and oftentimes deadly disease, but their prices are high, and payers may seek to limit spending. Total annual costs of covering all existing and expected CGTs for the entire US population 2023–2035 to amount to less than $20 per person and concentrate in commercial and state Medicaid
RENA M. CONTI +5 more
wiley +1 more source
Autologous and Heterologous Cell Therapy for Hemophilia B toward Functional Restoration of Factor IX
Cell Reports, 2018 Summary: Hemophilia B is an ideal target for gene- and cell-based therapies because of its monogenic nature and broad therapeutic index. Here, we demonstrate the use of cell therapy as a potential long-term cure for hemophilia B in our FIX-deficient ...
Suvasini Ramaswamy +7 more
doaj
Sharp Bounds for Neuman Means in Terms of Geometric, Arithemtic and Quadratic Means [PDF]
arXiv, 2014 In this paper, we find the greatest values $\alpha_{1}$, $\alpha_{2}$, $\alpha_{3}$, $\alpha_{4}$, $\alpha_{5}$, $\alpha_{6}$, $\alpha_{7}$, $\alpha_{8}$ and the least values $\beta_{1}$, $\beta_{2}$, $\beta_{3}$, $\beta_{4}$, $\beta_{5}$, $\beta_{6}$, $\beta_{7}$, $\beta_{8}$ such that the double inequalities $$A^{\alpha_{1}}(a,b)G^{1-\alpha_{1}}(a,b)<
arxiv
Blood, 2015 Emerging successful clinical data on gene therapy using adeno-associated viral (AAV) vector for hemophilia B (HB) showed that the risk of cellular immune response to vector capsid is clearly dose dependent. To decrease the vector dose, we explored AAV-8 (
J. Crudele +8 more
semanticscholar +1 more source
The Political Economy of the World Health Organization Model Lists of Essential Medicines
The Milbank Quarterly, Volume 103, Issue 1, Page 52-99, March 2025.Policy Points The World Health Organization (WHO) Model Lists of Essential Medicines (EML) aims to select clinically beneficial and cost‐effective medicines that ought to be prioritized by health systems based on the priority needs of their populations.
KRISTINA JENEI
wiley +1 more source
Patient Related Outcome Measures, 2019 Susan Cutter,1 Christine Guelcher,2 Susan Hunter,3 Dawn Rotellini,4 Spencer Dunn,5 David L Cooper61Penn Comprehensive Hemophilia and Thrombosis Program, Hospital of the University of Pennsylvania, Philadelphia, PA, USA; 2Comprehensive Hemostasis and ...
Cutter S +5 more
doaj
Evaluation of engineered AAV capsids for hepatic factor IX gene transfer in murine and canine models
Journal of Translational Medicine, 2017 Background Adeno-associated virus (AAV) gene therapy vectors have shown the best outcomes in human clinical studies for the treatment of genetic diseases such as hemophilia.
David M. Markusic +8 more
doaj +1 more source
Optimal bounds for a Gaussian Arithmetic-Geometric type mean by quadratic and contraharmonic means [PDF]
arXiv, 2018 In this paper, we present the best possible parameters $\alpha_i, \beta_i\ (i=1,2,3)$ and $\alpha_4,\beta_4\in(1/2,1)$ such that the double inequalities \begin{align*} \alpha_1Q(a,b)+(1-\alpha_1)C(a,b)&
arxiv
European Journal of Haematology, EarlyView.ABSTRACT Objective To report data from an ITI chart review study (NCT03951103) for first‐time and rescue ITI with recombinant factor VIII Fc fusion protein (rFVIIIFc) in persons with haemophilia A. Methods Retrospective and prospective real‐world data are reported from a non‐interventional, multicentre study of patients who had been or were currently ...
Robert Klamroth +7 more
wiley +1 more source
Refinements of the inequalities between Neuman-Sandor, arithmetic, contra-harmonic and quadratic means [PDF]
arXiv, 2012 In this paper, we prove that the inequalities $\alpha [1/3 Q(a,b)+2/3 A(a,b)]+(1-\alpha)Q^{1/3}(a,b)A^{2/3}(a,b)
arxiv