Nathalie Goemans Selected Research

eteplirsen

1/2022	A Combined Prospective and Retrospective Comparison of Long-Term Functional Outcomes Suggests Delayed Loss of Ambulation and Pulmonary Decline with Long-Term Eteplirsen Treatment.
1/2021	Comparison of Long-term Ambulatory Function in Patients with Duchenne Muscular Dystrophy Treated with Eteplirsen and Matched Natural History Controls.
1/2021	Open-Label Evaluation of Eteplirsen in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping: PROMOVI Trial.
1/2019	A Sequel to the Eteplirsen Saga: Eteplirsen Is Approved in the United States but Was Not Approved in Europe.
2/2016	Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.

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Nathalie Goemans Research Topics

Disease

32	Duchenne Muscular Dystrophy (Muscular Dystrophy, Becker) 04/2024 - 07/2005
6	Disease Progression 01/2023 - 08/2013
6	Muscular Diseases (Myopathy) 01/2022 - 10/2002
5	Spinal Muscular Atrophy (Progressive Muscular Atrophy) 05/2023 - 09/2020
4	Muscular Dystrophies (Muscular Dystrophy) 04/2024 - 10/2002
2	Vomiting 04/2024 - 01/2021
2	Headache (Headaches) 01/2021 - 05/2015
2	Ehlers-Danlos Syndrome (Syndrome, Ehlers-Danlos) 01/2020 - 05/2013
2	Injection Site Reaction 02/2019 - 01/2018
2	Rare Diseases (Rare Disease) 01/2016 - 10/2014
2	Hypertension (High Blood Pressure) 05/2015 - 05/2014
2	Weight Gain 05/2014 - 02/2010
2	Scoliosis 05/2014 - 10/2011
2	Minicore Myopathy with External Ophthalmoplegia 05/2004 - 10/2002
1	Adrenal Insufficiency (Adrenal Gland Hypofunction) 10/2022
1	Aphthous Stomatitis (Aphthous Ulcer) 01/2022
1	Arthralgia (Joint Pain) 01/2022
1	Exanthema (Rash) 01/2022
1	Fever (Fevers) 01/2022
1	Urinary Tract Infections (Urinary Tract Infection) 01/2022
1	Pneumonia (Pneumonitis) 01/2022
1	Cardiomyopathies (Cardiomyopathy) 01/2021
1	Hyperpigmentation 02/2019
1	Erythema 02/2019
1	Fibrosis (Cirrhosis) 02/2019
1	Nasopharyngitis 05/2015
1	Isolated Systolic Hypertension 05/2015
1	Inborn Genetic Diseases (Disease, Hereditary) 10/2014
1	Osteoporosis 05/2014
1	Cardiac Arrhythmias (Arrythmia) 05/2014
1	Dilated Cardiomyopathy (Cardiomyopathy, Congestive) 05/2014
1	Cataract (Cataracts) 05/2014
1	Cough 05/2014
1	Delayed Puberty 05/2014
1	Osteogenesis Imperfecta (Lobstein Disease) 05/2013
1	Overweight 02/2010
1	Neural Tube Defects (Exencephaly) 02/2010
1	Insulin Resistance 02/2010
1	Hemorrhage 02/2008
1	Mitochondrial Myopathies (Mitochondrial Myopathy) 08/2005
1	Bethlem myopathy 01/2005
1	Scleroatonic muscular dystrophy 01/2005
1	Spinal muscular atrophy with respiratory distress 1 09/2004
1	Desmin-Related Myofibrillar Myopathy 05/2004
1	Rigid spine syndrome 10/2002

Drug/Important Bio-Agent (IBA)

6	DystrophinIBA 04/2024 - 02/2019
5	Adrenal Cortex Hormones (Corticosteroids)IBA 04/2024 - 08/2013
5	eteplirsenIBA 01/2022 - 02/2016
5	idebenoneIBA 01/2017 - 06/2011
4	SteroidsIBA 10/2023 - 11/2007
4	RisdiplamIBA 05/2023 - 01/2022
4	GlucocorticoidsIBA 01/2022 - 09/2013
4	Nonsense Codon (Nonsense Mutation)IBA 01/2021 - 10/2014
3	atalurenIBA 10/2020 - 10/2014
3	CollagenIBA 01/2020 - 01/2005
3	PRO051IBA 02/2019 - 03/2017
2	Proteins (Proteins, Gene)FDA Link 01/2023 - 02/2019
2	Prednisone (Sone)FDA LinkGeneric 01/2023 - 10/2022
2	Antisense OligonucleotidesIBA 02/2019 - 01/2018
2	Selenoproteins (Selenoprotein)IBA 05/2004 - 10/2002
1	givinostatIBA 04/2024
1	Psychotropic Drugs (Psychoactive Drugs)IBA 07/2023
1	RNA Precursors (Precursor, mRNA)IBA 05/2023
1	deflazacortIBA 01/2023
1	Adrenocorticotropic Hormone (ACTH)FDA Link 10/2022
1	VBP15 compoundIBA 10/2022
1	Hydrocortisone (Cortisol)FDA LinkGeneric 10/2022
1	Biomarkers (Surrogate Marker)IBA 01/2022
1	Antisense RNA (Anti-Sense RNA)IBA 02/2019
1	OligonucleotidesIBA 02/2019
1	Tadalafil (Cialis)FDA Link 10/2017
1	MorpholinosIBA 02/2016
1	Investigational DrugsIBA 10/2014
1	Angiotensin-Converting Enzyme Inhibitors (ACE Inhibitors)IBA 05/2014
1	DiureticsIBA 05/2014
1	Glucose (Dextrose)FDA LinkGeneric 05/2014
1	Peptidyl-Dipeptidase A (Angiotensin Converting Enzyme)IBA 05/2014
1	Collagen Type I (Type I Collagen)IBA 05/2013
1	Peptide Hydrolases (Proteases)FDA Link 05/2013
1	Procollagen Type IIBA 05/2013
1	Metformin (Glucophage)FDA LinkGeneric 02/2010
1	Epoprostenol (Prostacyclin)FDA LinkGeneric 02/2008
1	oxatomideIBA 11/2007
1	Transfer RNA (tRNA)IBA 08/2005
1	Aspartic Acid (Aspartate)FDA Link 08/2005
1	CreatineIBA 07/2005
1	Glutamine (L-Glutamine)FDA Link 07/2005
1	Immunoglobulins (Immunoglobulin)IBA 09/2004
1	Carrier Proteins (Binding Protein)IBA 09/2004
1	DesminIBA 05/2004

Therapy/Procedure

7	Therapeutics 01/2022 - 07/2005
1	Spinal Fusion (Spondylosyndesis) 10/2011
1	Palliative Care (Palliative Therapy) 12/2010