Paula R Clemens Selected Research

Prednisone (Sone)

3/2024	Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial.
10/2022	Efficacy and Safety of Vamorolone vs Placebo and Prednisone Among Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
1/2020	Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study.

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Paula R Clemens Research Topics

Disease

33	Duchenne Muscular Dystrophy (Muscular Dystrophy, Becker) 03/2024 - 09/2002
5	Glycogen Storage Disease Type II (Pompe's Disease) 12/2023 - 04/2010
4	Disease Progression 01/2023 - 07/2013
3	Growth Disorders 03/2024 - 01/2016
3	Myositis (Idiopathic Inflammatory Myopathies) 01/2023 - 02/2011
3	Insulin Resistance 01/2019 - 01/2016
3	Necrosis 01/2018 - 09/2011
3	Inflammation (Inflammations) 08/2013 - 02/2011
2	Neoplasms (Cancer) 01/2018 - 12/2006
2	Cardiomyopathies (Cardiomyopathy) 08/2015 - 08/2014
2	Muscular Dystrophies (Muscular Dystrophy) 06/2015 - 10/2012
1	Adrenal Insufficiency (Adrenal Gland Hypofunction) 10/2022
1	Inborn Genetic Diseases (Disease, Hereditary) 01/2022
1	Myocardial Infarction 12/2021
1	Hirsutism 01/2020
1	Weight Gain 01/2020
1	Fibrosis (Cirrhosis) 01/2020
1	Bone Resorption 10/2018
1	Pneumonia (Pneumonitis) 01/2018
1	Proteinuria 05/2015
1	Spinal Muscular Atrophy (Progressive Muscular Atrophy) 11/2012
1	Osteoporosis 11/2012
1	Amyotrophic Lateral Sclerosis (Lou Gehrig's Disease) 11/2012
1	Scoliosis 11/2012
1	Chronic Disease (Chronic Diseases) 07/2012
1	Dilated Cardiomyopathy (Cardiomyopathy, Congestive) 03/2011
1	Muscular Diseases (Myopathy) 04/2010
1	Body Weight (Weight, Body) 04/2010
1	Cachexia 12/2006
1	Myoglobinuria 07/2003
1	Muscle Cramp (Cramp) 07/2003
1	Glycogen Storage Disease (Glycogenosis) 07/2003
1	Glycogen Storage Disease Type Ix 07/2003
1	Pain (Aches) 07/2003
1	Infections 05/2003
1	Myalgia 03/2002
1	Eosinophilia 03/2002

Drug/Important Bio-Agent (IBA)

12	DystrophinIBA 01/2023 - 09/2002
10	Proteins (Proteins, Gene)FDA Link 01/2023 - 05/2003
8	Biomarkers (Surrogate Marker)IBA 03/2024 - 06/2015
8	VBP15 compoundIBA 03/2024 - 10/2018
6	GlucocorticoidsIBA 01/2019 - 07/2013
4	viltolarsenIBA 01/2023 - 01/2020
4	EnzymesIBA 12/2021 - 03/2002
4	AcidsIBA 12/2021 - 04/2010
3	Prednisone (Sone)FDA LinkGeneric 03/2024 - 01/2020
3	GlucosidasesIBA 12/2021 - 11/2012
3	Peptides (Polypeptides)IBA 05/2015 - 05/2011
2	Histidine-tRNA LigaseIBA 01/2023 - 08/2013
2	AntigensIBA 01/2023 - 09/2002
2	human GAA protein (Myozyme)FDA Link 12/2021 - 04/2010
2	GlycogenIBA 12/2021 - 04/2010
2	Blood Proteins (Serum Proteins)IBA 01/2020 - 06/2015
2	Tumor Necrosis Factor-alpha (Tumor Necrosis Factor)IBA 01/2018 - 12/2006
2	CreatinineIBA 01/2016 - 05/2015
2	CreatineIBA 01/2016 - 07/2005
2	Protein Isoforms (Isoforms)IBA 05/2015 - 07/2003
2	Amino Acyl-tRNA Synthetases (Aminoacyl-tRNA Synthetase)IBA 08/2013 - 02/2011
1	Glucocorticoid Receptors (Glucocorticoid Receptor)IBA 03/2024
1	miglustat (Zavesca)FDA Link 12/2023
1	RNA (Ribonucleic Acid)IBA 01/2023
1	Adrenocorticotropic Hormone (ACTH)FDA Link 10/2022
1	Hydrocortisone (Cortisol)FDA LinkGeneric 10/2022
1	Adrenal Cortex Hormones (Corticosteroids)IBA 01/2020
1	deflazacortIBA 01/2020
1	SteroidsIBA 01/2020
1	LipopolysaccharidesIBA 01/2018
1	Phosphotransferases (Kinase)IBA 01/2018
1	Insulin (Novolin)FDA Link 01/2016
1	LeptinIBA 01/2016
1	SaltsIBA 01/2016
1	AngiotensinogenIBA 01/2016
1	somatotropin-binding protein (growth hormone-binding protein)IBA 01/2016
1	Therapeutic UsesIBA 11/2012
1	CalciumIBA 11/2012
1	Transcription Factors (Transcription Factor)IBA 07/2012
1	4- (4- dihexadecylaminostyryl)- N- methylpyridium (DiA)IBA 09/2011
1	Pentoxifylline (Trental)FDA LinkGeneric 08/2011
1	T-Cell Antigen Receptors (T-Cell Receptor)IBA 02/2011
1	Toll-Like Receptor 4IBA 02/2011
1	alpha-Glucosidases (Acid Maltase)IBA 04/2010
1	CytokinesIBA 12/2006
1	InterleukinsIBA 12/2006
1	NF-kappa B (NF-kB)IBA 12/2006
1	Conditioned Culture MediaIBA 12/2006
1	Glutamine (L-Glutamine)FDA Link 07/2005
1	Phosphorylase Kinase (Phosphorylase Kinase, Glycogen)IBA 07/2003
1	adenovirus receptorIBA 05/2003
1	IntegrinsIBA 05/2003

Therapy/Procedure

9	Therapeutics 12/2023 - 07/2005
1	Enzyme Replacement Therapy 12/2021
1	Heart Transplantation (Grafting, Heart) 03/2011