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Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease characterized by dysfunction and loss of motor neurons in the spinal cord, leading to progressive muscle atrophy, weakness, and paralysis.
FDA researchers recently released data on the long-term safety and response durability of patients after receiving Zolgensma treatment.
The START study is a phase I clinical study planned for 15 years.
Data collection work as of June 11, 2020, 13 children with a median age of 38.
All 10 patients in the treatment dose group survived without permanent ventilation.
Milestones after treatment
Milestones after treatmentThe results of this long-term clinical follow-up show that 5 years after receiving Zolgensma treatment, patients with type 1 SMA have long-term relief of clinical symptoms and have good long-term safety.
Five years after receiving Zolgensma treatment, the clinical symptoms of type 1 SMA patients have been relieved for a long time, and they have good long-term safety.
Original source:
Jerry R.
com/journals/jamaneurology/fullarticle/2780250" target="_blank" rel="noopener">Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy.
JAMA
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