Title | Journal | Journal Categories | Citations | Publication Date |
---|---|---|---|---|
Systematic Literature Review to Assess Economic Evaluations in Spinal Muscular Atrophy (SMA) | PharmacoEconomics |
| 8 | 2022 |
Onasemnogene abeparvovec for presymptomatic infants with two copies of SMN2 at risk for spinal muscular atrophy type 1: the Phase III SPR1NT trial | Nature Medicine |
| 101 | 2022 |
Newborn screening programs for spinal muscular atrophy worldwide: Where we stand and where to go | Neuromuscular Disorders |
| 94 | 2021 |
Spinal muscular atrophy: from rags to riches | Neuromuscular Disorders |
| 13 | 2021 |
Advances in treatment of spinal muscular atrophy—new phenotypes, new challenges, new implications for care | 2020 |