Ellen M Welch Selected Research

ataluren

7/2014	Ataluren for the treatment of nonsense-mutation cystic fibrosis: a randomised, double-blind, placebo-controlled phase 3 trial.
1/2013	Ataluren as an agent for therapeutic nonsense suppression.
2/2008	PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model.
5/2007	PTC124 targets genetic disorders caused by nonsense mutations.
4/2007	Safety, tolerability, and pharmacokinetics of PTC124, a nonaminoglycoside nonsense mutation suppressor, following single- and multiple-dose administration to healthy male and female adult volunteers.

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Ellen M Welch Research Topics

Disease

6	Inborn Genetic Diseases (Disease, Hereditary) 01/2017 - 04/2007
3	COVID-19 02/2022 - 08/2020
3	Cystic Fibrosis (Mucoviscidosis) 07/2014 - 04/2007
2	Spinal Muscular Atrophy (Progressive Muscular Atrophy) 05/2021 - 03/2016
2	Inflammation (Inflammations) 01/2021 - 08/2020
1	Ovarian Neoplasms (Ovarian Cancer) 01/2017
1	Neoplasms (Cancer) 01/2017
1	Muscular Atrophy (Muscle Atrophy) 03/2016
1	Acute Kidney Injury (Acute Renal Failure) 07/2014
1	Miyoshi myopathy 09/2010
1	type 2B Limb-girdle muscular dystrophy 09/2010
1	Headache (Headaches) 04/2007
1	Duchenne Muscular Dystrophy (Muscular Dystrophy, Becker) 04/2007
1	Dizziness (Lightheadedness) 04/2007

Drug/Important Bio-Agent (IBA)

6	Nonsense Codon (Nonsense Mutation)IBA 01/2017 - 04/2007
5	atalurenIBA 07/2014 - 04/2007
3	emvododstatIBA 02/2022 - 08/2020
3	Proteins (Proteins, Gene)FDA Link 07/2014 - 05/2007
2	CytokinesIBA 01/2021 - 08/2020
2	Pharmaceutical PreparationsIBA 01/2013 - 05/2007
1	Dihydroorotate DehydrogenaseIBA 02/2022
1	clitocineIBA 01/2017
1	MyostatinIBA 03/2016
1	ChloridesIBA 07/2014
1	Tobramycin (Nebcin)FDA LinkGeneric 07/2014
1	CreatinineIBA 07/2014
1	DysferlinIBA 09/2010
1	Terminator Codon (Termination Codon)IBA 02/2008
1	Messenger RNA (mRNA)IBA 04/2007
1	SuspensionsIBA 04/2007

Therapy/Procedure

5	Therapeutics 05/2021 - 07/2014
1	Intramuscular Injections 03/2016