Consensus from the Brazilian Academy of Neurology for the Diagnosis, Genetic Counseling, and Use of Disease-Modifying Therapies in 5q Spinal Muscular Atrophy
ARQUIVOS DE NEURO-PSIQUIATRIA(2024)
Key words
Spinal Muscular Atrophy,Survival of Motor Neuron 1 Protein,Oligonucleotides,Risdiplam,Genetic Therapy,Onasemnogene abeparvovec
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