Onasemnogene‐abeparvovec administration to premature infants with spinal muscular atrophy
SM Brown, AS Ajjarapu… - Annals of clinical …, 2024 - Wiley Online Library
Twin girls born at 30 weeks' gestation with spinal muscular atrophy (SMA) received
onsasemnogene‐abeparvovec (OA) at 3.5 weeks of life. They had no treatment‐related …
onsasemnogene‐abeparvovec (OA) at 3.5 weeks of life. They had no treatment‐related …
Long-read sequencing identifies copy-specific markers of SMN gene conversion in spinal muscular atrophy
MM Zwartkruis, MG Elferink, D Gommers, I Signoria… - medRxiv, 2024 - medrxiv.org
The complex 2 Mb survival motor neuron (SMN) locus on chromosome 5q13, including the
spinal muscular atrophy (SMA)-causing gene SMN1 and modifier SMN2, remains …
spinal muscular atrophy (SMA)-causing gene SMN1 and modifier SMN2, remains …