ABPMR Neuromuscular Medicine Practice Questions - ABPMR Neuromuscular Medicine Subspecialty Certifying Examination Exam

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A 6-month-old hypotonic infant has tongue fasciculations, absent reflexes, and paradoxical breathing. Genetic testing confirms homozygous SMN1 deletion with 2 copies of SMN2. Which therapy provides one-time IV gene replacement and is FDA-approved for SMA patients under 2 years of age?

Onasemnogene abeparvovec Nusinersen Risdiplam Tofersen

Correct Answer:

Onasemnogene abeparvovec

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