Long-term benefits of nusinersen in a child affected by cystic fibrosis and spinal muscular atrophy type 1

MeSH terms

• Child
• Cystic Fibrosis* / complications
• Cystic Fibrosis* / drug therapy
• Family
• Humans
• Muscular Atrophy, Spinal* / drug therapy
• Oligonucleotides
• Spinal Muscular Atrophies of Childhood* / drug therapy