Singer Jesy Nelson reveals her twins are unlikely to walk after diagnosis: 'I literally feel like my whole life has done a 360'
Singer's newborn twins were diagnosed with SMA1, received lifesaving treatment, and face ongoing hospital care with uncertain long-term motor outcomes.
My son was given world's most expensive gene therapy drug - now he can walk
A boy with spinal muscular atrophy treated with one-off gene therapy Zolgensma is now walking independently and achieving major developmental milestones.
Investigating New Treatments for Spinal Muscular Atrophy - News Center
Apitegromab showed encouraging safety and efficacy for improving motor function in nonambulatory children and adolescents with type 2 or 3 spinal muscular atrophy.