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Question 1 of 6

1. Question
Which of the following is NOT true regarding diagnosis of spinal muscular atrophy (SMA)?
- Multiplex ligation-dependent probe amplification (MLPA) is the gold standard for detection of homozygous deletions of exon 7 of survival of motor neuron 1 (SMN1)
- Initial diagnosis of SMA relies on electromyographic (EMG) assessment
- Survival of motor neuron 2 (SMN2) copy number is an important prognostic factor
- Neurofilament level in blood is a prognostic biomarker currently being studied
Question 2 of 6

2. Question
Which of the following is NOT true regarding medical treatment for SMA?
- The STR1VE-US trial showed an early Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) response
- The STR1VE-EU trial investigated infants with symptomatic SMA Type II
- The STR1VE-EU trial showed an early CHOP INTEND response
- The NURTURE, RAINBOWFISH, and SPR1NT trials investigate the importance of early intervention on treatment outcomes
Question 3 of 6

3. Question
Which of the following is NOT true regarding the SPR1NT trials?
- All patients with 2 copies of SMN2 showed improvement in Bayley Scales of Infant and Toddler Development (BSID) gross and fine motor scales
- All patients with 3 copies of SMN2 showed an improvement in BSID gross and fine motor scale
- Almost all patients with 2 copies of SMN2 achieved independent sitting for ≥ 30 seconds
- All patients with 3 copies of SMN2 achieved standing alone
Question 4 of 6

4. Question
Which of the following is NOT true regarding pulmonary care of patients with SMA?
- Secretion mobilization techniques for airway clearance include nebulisation of saline
- Recurrent chest infections are considered a supportive criterion for initiation of long-term non-invasive ventilation
- Recurrent chest infections are considered a major criterion for initiation of long-term non-invasive ventilation
- Maxillary hypoplasia is a potential complication of non-invasive ventilation
Question 5 of 6

5. Question
Which of the following is NOT true regarding the primary rehabilitation goals in SMA?
- In non-sitters, these include optimizing function, minimizing impairment, and optimizing tolerance to various positions
- In sitters, these include preventing contractures and scoliosis and maintaining, restoring, or promoting function and mobility
- In walkers, these include maintaining, restoring, or promoting function and mobility and adequate joint range, preventing contractures and scoliosis, and improving balance and endurance
- None of the above
Question 6 of 6

6. Question
Which of the following is NOT an SMN-dependent therapeutic strategy?
- Antisense oligonucleotide therapy
- Branaplam
- Onasemnogene abeparvovec-xioi
- Apitegromab

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