Baseline clinical and physical characteristics and of enrolled patients.

<p>Baseline clinical and physical characteristics and of enrolled patients.</p

Pulmonary function in SMA type 2 and 3 patients.

<p>Pulmonary function in SMA type 2 and 3 patients.</p

Electrophysiologic assessment of SMA type 2 and 3 patients.

<p>Electrophysiologic assessment of SMA type 2 and 3 patients.</p

Muscle MRI imaging of SMA type 2 and 3 patients.

<p>Muscle MRI imaging of SMA type 2 and 3 patients.</p

Motor function in SMA type 2 and 3 patients.

<p>Motor function in SMA type 2 and 3 patients.</p

Molecular SMA biomarkers.

<p>Molecular SMA biomarkers.</p

Psychomotor development of patients with SMA type 2 and 3.

<p>A: General motor development; B: Fine motor development; Values for ages displayed on histograms are median (min-max); * p ≤ 0.05.</p

CONSORT diagram.

<p>¹ As this manuscript was limited to baseline data, there were no withdrawal of participants. ² To be defined as “sitter” the patient must have a score ≥ 1 on item 9 of the MFM (“with support of one or both upper limbs maintain the seated position for 5 seconds”). For 3 patients the MFM could not be performed at baseline. 2 were classified as sitters since they had a score of 3 at the 6-month follow-up visit and 1 was retrospectively classified as a non-sitter according to patient files. ³ To be defined as “Ambulant” the patient must be able to walk 10 meters without human assistance or use of an ambulation device such as a cane or a walker.</p