Title: | Treating neonatal spinal muscular atrophy: A 21st century success story? |
Journal : | Early human development |
Authors: | Tizzano EF, Author |
Material Type: | Article |
Publisher: | Ireland, 10/2019 |
Languages: | English |
Keywords : | diagnosis ; early diagnosis ; medical treatment ; motor neuron disease ; neonatal screening ; neonatalogy ; neuromuscular disease ; presymptomatic stage ; review article ; SMN1-related proximal spinal muscular atrophy ; spinal muscular atrophy |
Keywords: | advanced therapies;antisense oligonucleotides;early diagnosis and intervention;gene therapy;genetic counselling;spinal muscular atrophy; |
Pubmed / DOI : | Pubmed : 31604576 / DOI : 10.1016/j.earlhumdev.2019.104851 |
N° Profil MNM : | 2019101 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/31604576 |