Biogen has announced that the first patient with spinal muscular atrophy (SMA) has been treated with Spinraza (nusinersen), who were prior treated with gene therapy Zolgensma (onasemnogene abeparvovec). The Phase 4 trial (RESPOND) is intended to determine the efficacy of Spinraza in infants and children who have still unmet need after treatment with Zolgensma. The study will be conducted at 20 sites in 60 children with SMA.
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Spinal muscular atrophy is a rare, genetic, neuromuscular disease. Children with SMA do not produce enough survival motor neuron (SMN) protein. This critical protein helps maintain motor neurons, neurons responsible for sitting, walking, and essential functions like breathing and swallowing.
RESPOND is a two-year, open-label study. Biogen announced that this trial helps in optimizing the treatment decisions. The Hammersmith Infant Neurological Examination Section 2 was the primary endpoint; safety, change from baseline on additional motor function measures, swallowing, and caretaker burden are secondary endpoints.
The study will enroll children who are considered to be appropriate for the Spinraza treatment by the investigator.
The primary group includes 40 infants, and the second group consists of 20 infants.