Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease.
N Engl J Med
; 381(17): 1644-1652, 2019 10 24.
Article
in En
| MEDLINE
| ID: mdl-31597037
Full text:
1
Database:
MEDLINE
Main subject:
Membrane Transport Proteins
/
Mutagenesis, Insertional
/
Oligonucleotides, Antisense
/
Rare Diseases
/
Precision Medicine
/
Neuronal Ceroid-Lipofuscinoses
Type of study:
Diagnostic_studies
/
Prognostic_studies
Limits:
Child
/
Female
/
Humans
Language:
En
Journal:
N Engl J Med
Year:
2019
Type:
Article