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description Publicationkeyboard_double_arrow_right Article 2016Ovid Technologies (Wolters Kluwer Health) NIH | Phase 2 Study of Mexileti..., NIH | Novel Molecular Mechanism..., CIHR +24 projectsNIH| Phase 2 Study of Mexiletine for the Treatment of Myotonic Dystrophy ,NIH| Novel Molecular Mechanisms of Neuromuscular Disease: Implications for Therapy ,CIHR ,NIH| BIOEQUIVALENCE AND CLINICAL IMPLICATIONS OF GENERIC BUPROPION ,NIH| IN VIVO NMR METABOLIC STUDY OF REGIONAL CARDIAC ISCHEMIA ,NIH| Evaluation of Treatments and Services to People with Duchenne Muscular Dystrophy ,NIH| BIOLOGY OF THE BONE MARROW DERIVED 3A1 STEM CELL ,NIH| Novel Strategy for Perioperative Beta-Blocker Therapy ,NIH| GENETICS-InFORMATICS TRIAL (GIFT) OF WARFARIN TO PREVENT DVT ,NIH| 9th International Myotonic Dystrophy Consortium Meeting ,NIH| Biiostatistics ,NIH| Disease Progression in Myotonic Dystrophy ,NIH| EXPERIMENTAL THERAPEUTICS IN NEUROLOGICAL DISEASE ,NIH| Comp A-NY State Surveillance and Research of MD and Neuromuscular Disorders ,NIH| BIOENERGETIC MECHANISMS ,NIH| POSITRON EMISSION TOMOGRAPHIC IMAGING OF LUNG TRANSPLANT ,SSHRC ,NIH| Phase 2 Study of 4-Aminopyridine for the Treatment of Episodic Ataxia Type 2 ,NIH| Remediating Age Related Cognitive Decline: Mindfulness-Based Stress Reduction and Exercise ,NIH| Genetically Informed Smoking Cessation Trial ,NIH| Ocular Hypertension Treatment Study 20-Year Follow-up: Clinical Center Grant ,NIH| RNA-mediated mechanisms in the myotonic dystrophies ,NIH| 7th International Myotonic Dystrophy Consortium Meeting ,NIH| FOR-DMD: Double-blind randomized trial to optimize steroid regimen in Duchenne MD ,NIH| Washington University Institute of Clinical and Translational Sciences ,NIH| Tenth International Myotonic Dystrophy Consortium Meeting ,NIH| ANALYSIS OF THE E COLI STB HEAT STABLE ENTEROTOXINAuthors: Griggs, Robert C.; Miller, J. Phillip; Greenberg, Cheryl R.; Fehlings, Darcy L.; +11 AuthorsGriggs, Robert C.; Miller, J. Phillip; Greenberg, Cheryl R.; Fehlings, Darcy L.; Pestronk, Alan; Mendell, Jerry R.; Moxley, Richard T.; King, Wendy; Kissel, John T.; Cwik, Valerie; Vanasse, Michel; Florence, Julaine M.; Pandya, Shree; Dubow, Jordan S.; Meyer, James M.;Objective: To assess safety and efficacy of deflazacort (DFZ) and prednisone (PRED) vs placebo in Duchenne muscular dystrophy (DMD). Methods: This phase III, double-blind, randomized, placebo-controlled, multicenter study evaluated muscle strength among 196 boys aged 5–15 years with DMD during a 52-week period. In phase 1, participants were randomly assigned to receive treatment with DFZ 0.9 mg/kg/d, DFZ 1.2 mg/kg/d, PRED 0.75 mg/kg/d, or placebo for 12 weeks. In phase 2, placebo participants were randomly assigned to 1 of the 3 active treatment groups. Participants originally assigned to an active treatment continued that treatment for an additional 40 weeks. The primary efficacy endpoint was average change in muscle strength from baseline to week 12 compared with placebo. The study was completed in 1995. Results: All treatment groups (DFZ 0.9 mg/kg/d, DFZ 1.2 mg/kg/d, and PRED 0.75 mg/kg/d) demonstrated significant improvement in muscle strength compared with placebo at 12 weeks. Participants taking PRED had significantly more weight gain than placebo or both doses of DFZ at 12 weeks; at 52 weeks, participants taking PRED had significantly more weight gain than both DFZ doses. The most frequent adverse events in all 3 active treatment arms were Cushingoid appearance, erythema, hirsutism, increased weight, headache, and nasopharyngitis. Conclusions: After 12 weeks of treatment, PRED and both doses of DFZ improved muscle strength compared with placebo. Deflazacort was associated with less weight gain than PRED. Classification of evidence: This study provides Class I evidence that for boys with DMD, daily use of either DFZ and PRED is effective in preserving muscle strength over a 12-week period.
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For further information contact us at helpdesk@openaire.eu115 citations 115 popularity Top 1% influence Top 10% impulse Top 1% Powered by BIP!visibility 2visibility views 2 download downloads 0 Powered bymore_vert add ClaimPlease grant OpenAIRE to access and update your ORCID works.This Research product is the result of merged Research products in OpenAIRE.
You have already added works in your ORCID record related to the merged Research product.This Research product is the result of merged Research products in OpenAIRE.
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For further information contact us at helpdesk@openaire.eudescription Publicationkeyboard_double_arrow_right Article 2019 EnglishBioMed Central NIH | Obesity Affects Immunity ..., SSHRC, NIH | Yale SPORE in Lung Cancer... +61 projectsNIH| Obesity Affects Immunity to Kidney Cancer ,SSHRC ,NIH| Yale SPORE in Lung Cancer (YSILC): The Biology and Personalized Treatment of Lung Cancer ,NIH| CANCER CENTER SUPPORT GRANT ,NIH| Oregon Clinical and Translational Research Institute TL1 Program ,NIH| TARGETED IMMUNOTHERAPY USING BISPECIFIC ANTIBODIES ,NIH| MOFFITT CANCER CENTER SUPPORT GRANT ,NIH| Immunoprevention and immunosurveillance of human non-viral cancers ,NIH| Cancer Immunology Training Program ,NIH| B7-H1/PD-1 modulation in cancer therapy ,NIH| Reactome: An Open Knowledgebase of Human Pathways ,NIH| Career Development Program ,NIH| Institutional Career Development Core ,UKRI| MICA: The North West England MRC Fellowship Scheme in Clinical Pharmacology and Therapeutics ,NIH| Nanoimmunoliposome-Complexed SPIO: Tumor-Specific Detection of Early Lung Cancer ,NIH| Treatment of glioblastoma using chain-like nanoparticles ,NIH| Cancer Cell Intrinsic Interferon-I pathway Activation by Fractionated Radiation ,NIH| Detection of micrometastasis using a dual-ligand nanoparticle ,NIH| Immuno-Oncology Translation Network: Data Management and Resource-Sharing Center at RPCI ,NIH| The Phenotypic Landscape of Cognitive Decline as Revealed by Next-Generation Multiplexed Ion Beam Imaging ,NIH| Winship Cancer Institute Cancer Center Support Grant ,NIH| Georgia Clinical & Translational Science Alliance (GaCTSA) ,NIH| LSUHSC-New Orleans Postbaccalaureate Research Education Program in Biomedical Sciences ,NIH| Clinical and Translational Science Award ,NIH| Ph1 Study of T-Vec given endoscopically for advanced pancreatic cancer IN 17248 (11/21/2016) ,NIH| PREDOCTORAL TRAINING IN CELL &MOLECULAR BIOLOGY ,NIH| Support of Yerkes National Primate Research Center ,NIH| Cancer Center Support (CORE) Grant ,NIH| Immunomodulation of the Tumor Microenvironment with Molecular Targeted Radiotherapy to Facilitate an Adaptive Anti-Tumor Immune Response to Combined Modality Immunotherapies ,NIH| CRCN: Dissecting Neural Circuits for Acute Pain ,NIH| Yale SPORE in Lung Cancer (YSILC): The Biology and Personalized Treatment of Lung Cancer ,NIH| Immune Monitoring and Analysis of Cancer at Stanford (IMACS) ,NIH| Developmental Funds ,NIH| Redox Biology and Medicine Training Program ,NIH| Redox Biology and Medicine Training Program ,NIH| Clinical and Translational Science Award ,NIH| MIRIAD - Multiplexed Imaging of Resilience In Alzheimers Disease ,NIH| Cancer Cell Intrinsic Interferon-I pathway Activation by Fractionated Radiation ,NIH| CANCER CENTER SUPPORT GRANT ,NIH| Therapuetic Immune Targeting of EphA2 Expressed by Melanoma &Its Tumor-Associate ,NIH| Targeting Transcriptional Regulators for Immunotherapy of Acute Myeloid Leukemia ,NIH| Epigenetic Modulation of Immune Tolerance by Ionizing Radiation and Chemotherapy ,NIH| Interdepartmental Biotechnology Training Program ,NIH| CANCER CENTER SUPPORT ,SNSF| Hyper-parameter 3D imaging of the bone marrow hematopoietic stem cell niche in leukemia and stress hematopoiesis by co-detection of expression (CODEX) ,SNSF| Deep phenotyping of the tumor microenvironment in hematological cancers by CODEX hyper-parameter tissue imaging ,NIH| Pediatric Scientist Development Program (K12) 2017-2022 ,NIH| Administrative Supplement to NIH-funded TL1 Training Grants ,NIH| Predictive signatures in breast cancer using multiplexed ion beam imaging ,NIH| Architecture and Trajectory of Acquired Resistance to Therapy in AML ,NIH| Project 1: Modeling tumor evolution in mouse and organoid models ,NIH| Research Training Program in Basic and Translational Oncology ,EC| PRE-DRIVE ,NIH| Shape Control and Transport Properties of DNA-Copolymer Micelles ,NIH| Biostatistics ,NIH| Moffitt Cancer Center Support Grant ,NIH| Therapeutic targeting of CD47 regulates tumor cell bioenergetics and mitophagy ,NIH| UAB Cancer Prevention and Control Training Program (T32) ,NIH| NRSA Training Core ,NIH| COMPREHENSIVE CANCER CENTER ,NIH| The Potential Role of Firboblast Activation Protein as a Natural Killer Cell Immune Checkpoint in Pancreatic Cancer ,NIH| Training in Cellular &Molecular Mechanisms of Tumor Rejection ,NIH| Effector mechanisms and therapeutic potential of MUC1 vaccine-elicited human antibodies ,NIH| MEDICAL SCIENTIST TRAINING PROGRAMAuthors: Orsolya Lorincz; Eszter Somogyi;Orsolya Lorincz; Eszter Somogyi;pmc: PMC6833180
pmid: 31694708
add ClaimPlease grant OpenAIRE to access and update your ORCID works.This Research product is the result of merged Research products in OpenAIRE.
You have already added works in your ORCID record related to the merged Research product.This Research product is the result of merged Research products in OpenAIRE.
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For further information contact us at helpdesk@openaire.eu25 citations 25 popularity Top 10% influence Average impulse Top 10% Powered by BIP!more_vert add ClaimPlease grant OpenAIRE to access and update your ORCID works.This Research product is the result of merged Research products in OpenAIRE.
You have already added works in your ORCID record related to the merged Research product.This Research product is the result of merged Research products in OpenAIRE.
You have already added works in your ORCID record related to the merged Research product.All Research productsarrow_drop_down <script type="text/javascript"> <!-- document.write('<div id="oa_widget"></div>'); document.write('<script type="text/javascript" src="https://www.openaire.eu/index.php?option=com_openaire&view=widget&format=raw&projectId=PMC6833180&type=result"></script>'); --> </script>
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description Publicationkeyboard_double_arrow_right Article 2016Ovid Technologies (Wolters Kluwer Health) NIH | Phase 2 Study of Mexileti..., NIH | Novel Molecular Mechanism..., CIHR +24 projectsNIH| Phase 2 Study of Mexiletine for the Treatment of Myotonic Dystrophy ,NIH| Novel Molecular Mechanisms of Neuromuscular Disease: Implications for Therapy ,CIHR ,NIH| BIOEQUIVALENCE AND CLINICAL IMPLICATIONS OF GENERIC BUPROPION ,NIH| IN VIVO NMR METABOLIC STUDY OF REGIONAL CARDIAC ISCHEMIA ,NIH| Evaluation of Treatments and Services to People with Duchenne Muscular Dystrophy ,NIH| BIOLOGY OF THE BONE MARROW DERIVED 3A1 STEM CELL ,NIH| Novel Strategy for Perioperative Beta-Blocker Therapy ,NIH| GENETICS-InFORMATICS TRIAL (GIFT) OF WARFARIN TO PREVENT DVT ,NIH| 9th International Myotonic Dystrophy Consortium Meeting ,NIH| Biiostatistics ,NIH| Disease Progression in Myotonic Dystrophy ,NIH| EXPERIMENTAL THERAPEUTICS IN NEUROLOGICAL DISEASE ,NIH| Comp A-NY State Surveillance and Research of MD and Neuromuscular Disorders ,NIH| BIOENERGETIC MECHANISMS ,NIH| POSITRON EMISSION TOMOGRAPHIC IMAGING OF LUNG TRANSPLANT ,SSHRC ,NIH| Phase 2 Study of 4-Aminopyridine for the Treatment of Episodic Ataxia Type 2 ,NIH| Remediating Age Related Cognitive Decline: Mindfulness-Based Stress Reduction and Exercise ,NIH| Genetically Informed Smoking Cessation Trial ,NIH| Ocular Hypertension Treatment Study 20-Year Follow-up: Clinical Center Grant ,NIH| RNA-mediated mechanisms in the myotonic dystrophies ,NIH| 7th International Myotonic Dystrophy Consortium Meeting ,NIH| FOR-DMD: Double-blind randomized trial to optimize steroid regimen in Duchenne MD ,NIH| Washington University Institute of Clinical and Translational Sciences ,NIH| Tenth International Myotonic Dystrophy Consortium Meeting ,NIH| ANALYSIS OF THE E COLI STB HEAT STABLE ENTEROTOXINAuthors: Griggs, Robert C.; Miller, J. Phillip; Greenberg, Cheryl R.; Fehlings, Darcy L.; +11 AuthorsGriggs, Robert C.; Miller, J. Phillip; Greenberg, Cheryl R.; Fehlings, Darcy L.; Pestronk, Alan; Mendell, Jerry R.; Moxley, Richard T.; King, Wendy; Kissel, John T.; Cwik, Valerie; Vanasse, Michel; Florence, Julaine M.; Pandya, Shree; Dubow, Jordan S.; Meyer, James M.;Objective: To assess safety and efficacy of deflazacort (DFZ) and prednisone (PRED) vs placebo in Duchenne muscular dystrophy (DMD). Methods: This phase III, double-blind, randomized, placebo-controlled, multicenter study evaluated muscle strength among 196 boys aged 5–15 years with DMD during a 52-week period. In phase 1, participants were randomly assigned to receive treatment with DFZ 0.9 mg/kg/d, DFZ 1.2 mg/kg/d, PRED 0.75 mg/kg/d, or placebo for 12 weeks. In phase 2, placebo participants were randomly assigned to 1 of the 3 active treatment groups. Participants originally assigned to an active treatment continued that treatment for an additional 40 weeks. The primary efficacy endpoint was average change in muscle strength from baseline to week 12 compared with placebo. The study was completed in 1995. Results: All treatment groups (DFZ 0.9 mg/kg/d, DFZ 1.2 mg/kg/d, and PRED 0.75 mg/kg/d) demonstrated significant improvement in muscle strength compared with placebo at 12 weeks. Participants taking PRED had significantly more weight gain than placebo or both doses of DFZ at 12 weeks; at 52 weeks, participants taking PRED had significantly more weight gain than both DFZ doses. The most frequent adverse events in all 3 active treatment arms were Cushingoid appearance, erythema, hirsutism, increased weight, headache, and nasopharyngitis. Conclusions: After 12 weeks of treatment, PRED and both doses of DFZ improved muscle strength compared with placebo. Deflazacort was associated with less weight gain than PRED. Classification of evidence: This study provides Class I evidence that for boys with DMD, daily use of either DFZ and PRED is effective in preserving muscle strength over a 12-week period.
add ClaimPlease grant OpenAIRE to access and update your ORCID works.This Research product is the result of merged Research products in OpenAIRE.
You have already added works in your ORCID record related to the merged Research product.This Research product is the result of merged Research products in OpenAIRE.
You have already added works in your ORCID record related to the merged Research product.All Research productsarrow_drop_down <script type="text/javascript"> <!-- document.write('<div id="oa_widget"></div>'); document.write('<script type="text/javascript" src="https://www.openaire.eu/index.php?option=com_openaire&view=widget&format=raw&projectId=10.1212/wnl.0000000000003217&type=result"></script>'); --> </script>
For further information contact us at helpdesk@openaire.eu115 citations 115 popularity Top 1% influence Top 10% impulse Top 1% Powered by BIP!visibility 2visibility views 2 download downloads 0 Powered bymore_vert add ClaimPlease grant OpenAIRE to access and update your ORCID works.This Research product is the result of merged Research products in OpenAIRE.
You have already added works in your ORCID record related to the merged Research product.This Research product is the result of merged Research products in OpenAIRE.
You have already added works in your ORCID record related to the merged Research product.All Research productsarrow_drop_down <script type="text/javascript"> <!-- document.write('<div id="oa_widget"></div>'); document.write('<script type="text/javascript" src="https://www.openaire.eu/index.php?option=com_openaire&view=widget&format=raw&projectId=10.1212/wnl.0000000000003217&type=result"></script>'); --> </script>
For further information contact us at helpdesk@openaire.eudescription Publicationkeyboard_double_arrow_right Article 2019 EnglishBioMed Central NIH | Obesity Affects Immunity ..., SSHRC, NIH | Yale SPORE in Lung Cancer... +61 projectsNIH| Obesity Affects Immunity to Kidney Cancer ,SSHRC ,NIH| Yale SPORE in Lung Cancer (YSILC): The Biology and Personalized Treatment of Lung Cancer ,NIH| CANCER CENTER SUPPORT GRANT ,NIH| Oregon Clinical and Translational Research Institute TL1 Program ,NIH| TARGETED IMMUNOTHERAPY USING BISPECIFIC ANTIBODIES ,NIH| MOFFITT CANCER CENTER SUPPORT GRANT ,NIH| Immunoprevention and immunosurveillance of human non-viral cancers ,NIH| Cancer Immunology Training Program ,NIH| B7-H1/PD-1 modulation in cancer therapy ,NIH| Reactome: An Open Knowledgebase of Human Pathways ,NIH| Career Development Program ,NIH| Institutional Career Development Core ,UKRI| MICA: The North West England MRC Fellowship Scheme in Clinical Pharmacology and Therapeutics ,NIH| Nanoimmunoliposome-Complexed SPIO: Tumor-Specific Detection of Early Lung Cancer ,NIH| Treatment of glioblastoma using chain-like nanoparticles ,NIH| Cancer Cell Intrinsic Interferon-I pathway Activation by Fractionated Radiation ,NIH| Detection of micrometastasis using a dual-ligand nanoparticle ,NIH| Immuno-Oncology Translation Network: Data Management and Resource-Sharing Center at RPCI ,NIH| The Phenotypic Landscape of Cognitive Decline as Revealed by Next-Generation Multiplexed Ion Beam Imaging ,NIH| Winship Cancer Institute Cancer Center Support Grant ,NIH| Georgia Clinical & Translational Science Alliance (GaCTSA) ,NIH| LSUHSC-New Orleans Postbaccalaureate Research Education Program in Biomedical Sciences ,NIH| Clinical and Translational Science Award ,NIH| Ph1 Study of T-Vec given endoscopically for advanced pancreatic cancer IN 17248 (11/21/2016) ,NIH| PREDOCTORAL TRAINING IN CELL &MOLECULAR BIOLOGY ,NIH| Support of Yerkes National Primate Research Center ,NIH| Cancer Center Support (CORE) Grant ,NIH| Immunomodulation of the Tumor Microenvironment with Molecular Targeted Radiotherapy to Facilitate an Adaptive Anti-Tumor Immune Response to Combined Modality Immunotherapies ,NIH| CRCN: Dissecting Neural Circuits for Acute Pain ,NIH| Yale SPORE in Lung Cancer (YSILC): The Biology and Personalized Treatment of Lung Cancer ,NIH| Immune Monitoring and Analysis of Cancer at Stanford (IMACS) ,NIH| Developmental Funds ,NIH| Redox Biology and Medicine Training Program ,NIH| Redox Biology and Medicine Training Program ,NIH| Clinical and Translational Science Award ,NIH| MIRIAD - Multiplexed Imaging of Resilience In Alzheimers Disease ,NIH| Cancer Cell Intrinsic Interferon-I pathway Activation by Fractionated Radiation ,NIH| CANCER CENTER SUPPORT GRANT ,NIH| Therapuetic Immune Targeting of EphA2 Expressed by Melanoma &Its Tumor-Associate ,NIH| Targeting Transcriptional Regulators for Immunotherapy of Acute Myeloid Leukemia ,NIH| Epigenetic Modulation of Immune Tolerance by Ionizing Radiation and Chemotherapy ,NIH| Interdepartmental Biotechnology Training Program ,NIH| CANCER CENTER SUPPORT ,SNSF| Hyper-parameter 3D imaging of the bone marrow hematopoietic stem cell niche in leukemia and stress hematopoiesis by co-detection of expression (CODEX) ,SNSF| Deep phenotyping of the tumor microenvironment in hematological cancers by CODEX hyper-parameter tissue imaging ,NIH| Pediatric Scientist Development Program (K12) 2017-2022 ,NIH| Administrative Supplement to NIH-funded TL1 Training Grants ,NIH| Predictive signatures in breast cancer using multiplexed ion beam imaging ,NIH| Architecture and Trajectory of Acquired Resistance to Therapy in AML ,NIH| Project 1: Modeling tumor evolution in mouse and organoid models ,NIH| Research Training Program in Basic and Translational Oncology ,EC| PRE-DRIVE ,NIH| Shape Control and Transport Properties of DNA-Copolymer Micelles ,NIH| Biostatistics ,NIH| Moffitt Cancer Center Support Grant ,NIH| Therapeutic targeting of CD47 regulates tumor cell bioenergetics and mitophagy ,NIH| UAB Cancer Prevention and Control Training Program (T32) ,NIH| NRSA Training Core ,NIH| COMPREHENSIVE CANCER CENTER ,NIH| The Potential Role of Firboblast Activation Protein as a Natural Killer Cell Immune Checkpoint in Pancreatic Cancer ,NIH| Training in Cellular &Molecular Mechanisms of Tumor Rejection ,NIH| Effector mechanisms and therapeutic potential of MUC1 vaccine-elicited human antibodies ,NIH| MEDICAL SCIENTIST TRAINING PROGRAMAuthors: Orsolya Lorincz; Eszter Somogyi;Orsolya Lorincz; Eszter Somogyi;pmc: PMC6833180
pmid: 31694708
add ClaimPlease grant OpenAIRE to access and update your ORCID works.This Research product is the result of merged Research products in OpenAIRE.
You have already added works in your ORCID record related to the merged Research product.This Research product is the result of merged Research products in OpenAIRE.
You have already added works in your ORCID record related to the merged Research product.All Research productsarrow_drop_down <script type="text/javascript"> <!-- document.write('<div id="oa_widget"></div>'); document.write('<script type="text/javascript" src="https://www.openaire.eu/index.php?option=com_openaire&view=widget&format=raw&projectId=PMC6833180&type=result"></script>'); --> </script>
For further information contact us at helpdesk@openaire.eu25 citations 25 popularity Top 10% influence Average impulse Top 10% Powered by BIP!more_vert add ClaimPlease grant OpenAIRE to access and update your ORCID works.This Research product is the result of merged Research products in OpenAIRE.
You have already added works in your ORCID record related to the merged Research product.This Research product is the result of merged Research products in OpenAIRE.
You have already added works in your ORCID record related to the merged Research product.All Research productsarrow_drop_down <script type="text/javascript"> <!-- document.write('<div id="oa_widget"></div>'); document.write('<script type="text/javascript" src="https://www.openaire.eu/index.php?option=com_openaire&view=widget&format=raw&projectId=PMC6833180&type=result"></script>'); --> </script>
For further information contact us at helpdesk@openaire.eu