1
TITLE: Theranostics vs theratyping or theranostics plus theratyping?  Full Text
AUTHORS: Amaral, Margarida D.; Pankonien, Ines;
PUBLISHED: 2025, SOURCE: JOURNAL OF CYSTIC FIBROSIS, VOLUME: 24, ISSUE: 1
INDEXED IN: Scopus WOS CrossRef
IN MY: ORCID
2
TITLE: The role of CFTR in the eye, and the effect of early highly effective modulator treatment for cystic fibrosis on eye health
AUTHORS: Schneider Futschik, Elena K.; Zhu, Yimin; Li, Danni; Habgood, Mark D.; Nguyen, Bao N.; Pankonien, Ines; Amaral, Margarida D.; Downie, Laura E.; Chinnery, Holly R.;
PUBLISHED: 2024, SOURCE: PROGRESS IN RETINAL AND EYE RESEARCH, VOLUME: 103
INDEXED IN: Scopus WOS CrossRef
IN MY: ORCID
3
TITLE: What Can RNA-Based Therapy Do for Monogenic Diseases?
AUTHORS: Clarke, Luka A.; Amaral, Margarida D.;
PUBLISHED: 2023, SOURCE: PHARMACEUTICS, VOLUME: 15, ISSUE: 1
INDEXED IN: Scopus WOS CrossRef: 2
IN MY: ORCID
4
TITLE: Human epididymis protein 4 (HE4) plasma concentration inversely correlates with the improvement of cystic fibrosis lung disease in p.Phe508del-CFTR homozygous cases treated with the CFTR modulator <i>lumacaftor/ivacaftor</i> combination  Full Text
AUTHORS: Pocsi, Marianna; Fejes, Zsolt; Bene, Zsolt; Nagy, Attila; Balogh, Istvan; Amaral, Margarida D.; Macek Jr, Milan; Nagy Jr, Bela;
PUBLISHED: 2023, SOURCE: JOURNAL OF CYSTIC FIBROSIS, VOLUME: 22, ISSUE: 6
INDEXED IN: WOS
5
TITLE: Precision medicine for rare diseases: The times they are A-Changin'
AUTHORS: Amaral, Margarida D.;
PUBLISHED: 2022, SOURCE: CURRENT OPINION IN PHARMACOLOGY, VOLUME: 63
INDEXED IN: Scopus WOS CrossRef: 7
IN MY: ORCID
6
TITLE: Using the genome to correct the ion transport defect in cystic fibrosis
AUTHORS: Amaral, Margarida D.;
PUBLISHED: 2022, SOURCE: JOURNAL OF PHYSIOLOGY-LONDON, VOLUME: 601, ISSUE: 9
INDEXED IN: Scopus WOS CrossRef: 1
IN MY: ORCID
7
TITLE: How to determine the mechanism of action of CFTR modulator compounds: A gateway to theranostics
AUTHORS: Amaral, MD;
PUBLISHED: 2021, SOURCE: EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY, VOLUME: 210
INDEXED IN: Scopus WOS CrossRef: 8
IN MY: ORCID
8
TITLE: Activating alternative chloride channels to treat CF: Friends or Foes? Report on the Meeting of the Basic Science Working Group in Dubrovnik, Croatia
AUTHORS: Amaral, MD; Beekman, JM;
PUBLISHED: 2020, SOURCE: JOURNAL OF CYSTIC FIBROSIS, VOLUME: 19, ISSUE: 1
INDEXED IN: Scopus WOS
IN MY: ORCID
9
TITLE: CFTR processing, trafficking and interactions  Full Text
AUTHORS: Amaral, MD; Hutt, DM; Tomati, V; Botelho, HM; Pedemonte, N;
PUBLISHED: 2020, SOURCE: JOURNAL OF CYSTIC FIBROSIS, VOLUME: 19
INDEXED IN: Scopus WOS CrossRef: 18
IN MY: ORCID
10
TITLE: A central role of the endoplasmic reticulum in the cell emerges from its functional contact sites with multiple organelles
AUTHORS: Almeida, C; Amaral, MD;
PUBLISHED: 2020, SOURCE: CELLULAR AND MOLECULAR LIFE SCIENCES, VOLUME: 77, ISSUE: 23
INDEXED IN: Scopus WOS CrossRef: 17
IN MY: ORCID
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