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Investigational gene therapy
DFT383 is an investigational gene therapy under development by Novartis for the treatment of cystinosis, a rare autosomal recessive lysosomal storage disorder
DFT383
Lysosomal storage disease
thereby reducing cystine accumulation in various tissues. AVR-RD-04 (now DFT383 by Novartis) This investigational gene therapy, initially developed by AVROBIO
Cystinosis
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Girl/Female
Indian
To be Carefree
Girl/Female
Arabic, Australian, Malaysian
Onlooker
Boy/Male
English
Maker of rope.
Girl/Female
Arabic
The Beauty of Eyes
Girl/Female
Arabic, Hebrew
Rocket
Boy/Male
Hindu
Lord Krishna
Boy/Male
Hindu, Indian, Sanskrit
Inducing Pride
Boy/Male
Arabic
Servant of the Capable
Male
English
Anglicized form of Hebrew Yeshayah, JESAIAH means "God is salvation." In the bible, this is the name of one of the most famous prophets. Also spelled Isaiah and Jeshaiah.
Boy/Male
Norse
Eagle spear.
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