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PTC 124

Known as: PTC-124, PTC124 
National Institutes of Health

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Papers overview

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2019
2019
Readthrough Intervention Increases ER Stress in Wolfram Syndrome
Aim: Wolfram syndrome (WFS) is an example of inherited endocrine and neurodegenerative disease due to increased ER stress with… 
Review
2016
Review
2016
Molecular therapies for Duchenne muscular dystrophy.
Duchenne muscular dystrophy (DMD) is the most common inherited muscle disorder, which is characterized by progressive muscle… 
2016
2016
The effect of G418 and PTC124 as suppression therapy for Beta Thalassemia
Tese de mestrado, Biologia Molecular e Genetica, Universidade de Lisboa, Faculdade de Ciencias, 2016 
Review
2015
Review
2015
[Genetic Diagnosis and Molecular Therapies for Duchenne Muscular Dystrophy].
Duchenne muscular dystrophy (DMD) is the most common form of inherited muscle disease and is characterized by progressive muscle… 
2014
2014
Families’ experiences with medical research for pediatric rare diseases : a qualitative ethnographic study of parents and children participating in clinical trials for Duchenne muscular dystrophy…
The biopharmaceutical industry has recently expanded its focus on developing new cures for rare diseases. As a growing number of… 
2013
2013
PTC124 DERIVATIVES AS A NOVEL APPROACH TO IMPROVE THE READTHROUGH OF PREMATURE AMBER AND OCHRE STOP CODONS
2011
2011
PTC124 derivatives as a novel approach to improve the readthrough of premature stop codons in the CFTR gene.