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Orphanet Journal of Rare Diseases|May 22, 2021
Enzyme replacement therapy and hematopoietic stem cell transplant: a new paradigm of treatment in Wolman diseaseJane E Potter, Gemma Petts, Arunabha Ghosh, et al.
Brain : a Journal of Neurology|January 9, 2015
Neuroinflammation, mitochondrial defects and neurodegeneration in mucopolysaccharidosis III type C mouse modelCarla Martins, Helena Hůlková, Larbi Dridi, et al.
Circulation. Cardiovascular Quality and Outcomes|February 12, 2015
Survival benefit of the primary prevention implantable cardioverter-defibrillator among older patients: does age matter? An analysis of pooled data from 5 clinical trialsPaul L Hess, Sana M Al-Khatib, Joo Y Han, et al.
Biorxiv : the Preprint Server for Biology|May 7, 2024
Secondary deficiency of neuraminidase 1 contributes to CNS pathology in neurological mucopolysaccharidoses via hypersialylation of brain glycoproteinsTianMeng Xu, Rachel Heon-Roberts, Travis Moore, et al.
JACC. Heart Failure|October 13, 2014
Outcomes of implantable cardioverter-defibrillator use in patients with comorbidities: results from a combined analysis of 4 randomized clinical trialsBenjamin A Steinberg, Sana M Al-Khatib, Rex Edwards, et al.
The Journal of Steroid Biochemistry and Molecular Biology|November 27, 2020
Bile acid biosynthesis in Smith-Lemli-Opitz syndrome bypassing cholesterol: Potential importance of pathway intermediatesJonas Abdel-Khalik, Thomas Hearn, Alison L Dickson, et al.
The New England Journal of Medicine|March 16, 2010
Effects of intensive blood-pressure control in type 2 diabetes mellitus, William C Cushman, Gregory W Evans, et al.
Contemporary Clinical Trials|May 30, 2012
Design and baseline data from the vanguard of the Comparison of Depression Interventions after Acute Coronary Syndrome (CODIACS) randomized controlled trialWilliam Whang, Matthew M Burg, Robert M Carney, et al.
The New England Journal of Medicine|March 16, 2010
Effects of combination lipid therapy in type 2 diabetes mellitus, Henry N Ginsberg, Marshall B Elam, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|August 9, 2005
Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal miceMike Themis, Simon N Waddington, Manfred Schmidt, et al.
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