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Efficacy Evaluation of Liver-Directed Gene Therapy in Fabry Mice
Conclusions: Collectively, these data provide strong evidence that our liver-directed AAV-mediated gene therapy approach holds considerable therapeutic potential for the treatment of Fabry disease. We anticipate that a single dose IV procedure will pose minimal burden to Fabry patients and will be a viable alternative to biweekly enzyme infusions, potentially reducing treatment-related morbidity whislt improving patient quality of life and potentially providing them with a functional long-term cure.DisclosuresKia: Freeline: Employment, Equity Ownership. McIntosh: Freeline: Consultancy. Hosseini: Freeline: Employment, Equit...
Source: Blood - November 21, 2018 Category: Hematology Authors: Kia, A., McIntosh, J., Rosales, C., Hosseini, P., Sheridan, R., Spiewak, J., Mills, K., Corbau, R., Nathwani, A. C. Tags: 801. Gene Therapy and Transfer: Poster I Source Type: research

Final Results from a Phase I Trial Combining Selinexor with High-Dose Cytarabine (HiDAC) and Mitoxantrone (Mito) for Remission Induction in Acute Myeloid Leukemia (AML)
Conclusions: The selinexor/HiDAC/Mito regimen is feasible and tolerable at selinexor doses up to 80mg/day or ~50 mg/m2/day twice weekly. This regimen yields an ORR of 64% based on currently available data. We had previously reported molecular correlatives demonstrating the effect of selinexor. The recommended phase 2 dose is 80mg of selinexor.Figure.DisclosuresLarson: Ariad/Takeda: Consultancy, Research Funding; BristolMyers Squibb: Consultancy, Research Funding; Novartis: Consultancy, Research Funding; Pfizer: Consultancy, Research Funding. Odenike: Agios: Research Funding; Astex: Research Funding; Dava Oncology: Consulta...
Source: Blood - November 21, 2018 Category: Hematology Authors: Wang, A., Weiner, H., Larson, R. A., Odenike, O., Artz, A. S., Bishop, M. R., Godley, L., Thirman, M., Kosuri, S., Churpek, J., Curran, E. K., Pettit, K., Stock, W., Liu, H. Tags: 616. Acute Myeloid Leukemia: Novel Therapy, excluding Transplantation: Poster III Source Type: research

DIS-17-0023 The Enduring Health Challenges of Afghan Immigrants and Refugees in Iran: A Systematic Review
This article does not contain any studies with human participants or animals performed by the author. Data Availability All national (MagIran, Science Information Database (SID) and Iranmedex) and international (PubMed, Scopus) databases were searched from November 2010 to November 2016 using keywords both in English and Persian: Afghan immigrants, Afghan refugees, Iran, infectious diseases, tuberculosis, HIV, Hepatitis B and C, non-communicable disease, food security, mental health, barriers, health insurance, access to health service. All related websites and webpages were also searched by Google with the same keywords ...
Source: PLOS Currents Disasters - July 21, 2017 Category: International Medicine & Public Health Authors: nasim Source Type: research

The Enduring Health Challenges of Afghan Immigrants and Refugees in Iran: A Systematic Review
This article does not contain any studies with human participants or animals performed by the author. Data Availability All national (MagIran, Science Information Database (SID) and Iranmedex) and international (PubMed, Scopus) databases were searched from November 2010 to November 2016 using keywords both in English and Persian: Afghan immigrants, Afghan refugees, Iran, infectious diseases, tuberculosis, HIV, Hepatitis B and C, non-communicable disease, food security, mental health, barriers, health insurance, access to health service. All related websites and webpages were also searched by Google with the same keywords ...
Source: PLOS Currents Disasters - July 21, 2017 Category: International Medicine & Public Health Authors: nasim Source Type: research