Individual details of imaging and PUL findings at shoulder, arm and forearm level.

<p>The shading reflects the severity of involvement with the score of 0 shown as a white cell, score of 1 as pale green, score of 2 as yellow, score of 3 as orange and score of 4 as red. The grade 2.5 was used to identify patients with 2b involvement. Del = deltoid; suprasp = supraspinatus; infrasp = infraspinatus; subscap = subscapularis; pec = pectoralis; corac = coraco-brachialis; serr = serratus anterior; lat = latissimus dorsi; bic = biceps brachii; brac = brachialis;tri = triceps brachii; sup = supinator;pron = pronator teres;F cp = flexor carpi radialis; palm = palmar; F ds = flexor digitorum superficialis; F cu = flexor carpi ulnaris; Fdp = flexor digitorum profundus; Anc = anconeus; E cu = extensor carpi ulnaris; Edm = extensor digiti minimi; E d = extensor digitorum; E cr = extensor carpi radialis; Br R = brachioradialis; F pl = flexor pollicis longus; E pl = extensor pollicis longus. Ambulant (E) = ambulant early; Ambulant (L): ambulant late; Non ambulant (E) = non ambulant early; Non ambulant (L): non ambulant late.</p

T1-weighted axial MRI of the forearm.

<p>T1-weighted axial MRI of the forearm at the level of the radial tuberosity showing increased signal suggestive of fatty replacement in the forearm muscles, especially in the supinator (arrow) and pronator teres (arrowhead). The involvement is milder in the youngest ambulant patients aged 8 and 14 (a and b respectively) and more marked in the older non-ambulant ones, both aged 17 (c and d), with severe involvement shown in image d. R (radius); U (ulna).</p

PUL and patients age.

<p>Details of the PUL scores according to age.</p

T1-weighted axial MRI of the arm.

<p>T1-weighted axial MRI of the arm at the mid-level of the humeral shaft (h) showing progressive increased signal suggestive of fatty replacement in the arm muscles. The involvement is milder in the youngest ambulant patients aged 5 and 11 (a and b respectively) and more marked in the older non-ambulant ones, both 17 years old (c and d) with severe involvement of all the muscles shown in image d.</p

T1-weighted axial MRI of the shoulder.

<p>T1-weighted axial MRI of the shoulder at the level of the humeral head (H) showing increased signal suggestive of fatty replacement in the muscles at this level. The involvement is milder in the youngest ambulant patients aged 10 and 11 (a and b respectively) with more evident fatty replacement in the subscapularis (arrowhead) and infraspinatus (arrow) and more marked in the older non-ambulant ones, both 17 years old (c and d), with severe involvement of all the muscles shown in image d.</p

Pul and MRI total scores.

<p>Details of the PUL and MRI total scores.</p

Pul entry items and MRI total scores.

<p>Details of the PUL entry items and MRI total scores. The entry items range from score 0 –no useful hand function—to score 6 full shoulder abduction–no weakness.</p

Revised North Star Ambulatory Assessment for Young Boys with Duchenne Muscular Dystrophy

<div><p>The advent of therapeutic approaches for Duchenne muscular dystrophy (DMD) has highlighted the need to identify reliable outcome measures for young boys with DMD. The aim of this study was to develop a revised version of the North Star Ambulatory Assessment (NSAA) suitable for boys between the age of 3 and 5 years by identifying age appropriate items and revising the scoring system accordingly. Using the scale in 171 controls between the age of 2.9 and 4.8 years, we identified items that were appropriate at different age points. An item was defined as age appropriate if it was completed, achieving a full score, by at least 85% of the typically developing boys at that age. At 3 years (±3months) there were only 8 items that were age appropriate, at 3 years and 6 months there were 13 items while by the age of 4 years all 17 items were appropriate. A revised version of the scale was developed with items ordered according to the age when they could be reliably performed. The application of the revised version of the scale to data collected in young DMD boys showed that very few of the DMD boys were able to complete with a full score all the age appropriate items. In conclusion, our study suggests that a revised version of the NSAA can be used in boys from the age of 3 years to obtain information on how young DMD boys acquire new abilities and how this correlates with their peers.</p></div

Revised version of the NSAA with items ordered according to the age when they can be performed.

<p>Revised version of the NSAA with items ordered according to the age when they can be performed.</p

Frequency distribution of controls reaching a full score on individual NSAA items at different age points.

<p>The shaded cells indicate the activities that were achieved by at least 85% of the boys at that age point and were therefore considered as age appropriate.</p