Results 201 to 210 of about 19,843 (235)
Some of the next articles are maybe not open access.
Risdiplam-Treated Infants with Type 1 Spinal Muscular Atrophy versus Historical Controls
New England Journal of Medicine, 2021Riccardo Masson +2 more
exaly
The genetic heterogeneity of spinal muscular atrophy (SMA).
Birth defects original article series, 1975The clinical picture of the spinal muscular atrophy varies greatly with respect to age of onset, speed of progression, severity and distribution of muscular atrophy, weakness and contractures, yet cases occurring within a family usually show concordant clinical features. Thus, genetic heterogeneity has to be assumed.
openaire +1 more source
Risdiplam in Type 1 Spinal Muscular Atrophy
New England Journal of Medicine, 2021Giovanni Baranello +2 more
exaly
Base editing rescue of spinal muscular atrophy in cells and in mice
Science, 2023Mandana Arbab +2 more
exaly