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Spinal Muscular Atrophy III (Kugelberg-Weilander Syndrome)

In more chronic SMA III, also referred to as Kugelberg- Welander syndrome, weakness usually initially occurs between the ages of 18 months and late teens. Motor milestones may be delayed in infancy.

Proximal weak­ness is observed, with the pelvic girdle being more affected than the shoulder girdle (57). There is an exaggerated lumbar lordosis and anterior pelvic tilt owing to hip extensor weakness. There is also a wad­dling gait pattern with pelvic drop and lateral trunk lean over the stance-phase side, secondary to hip abductor weakness. If ankle plantar flexion strength is sufficient, the patients may show primarily forefoot or toe contact and no heel strike similar to patients with Duchenne dystrophy. This is a compensatory measure for knee extensor weakness to maintain a stabilizing knee extension moment at the knee. The patient may exhibit a Gower's sign when arising from the floor; stair climbing is difficult due to hip flexor weakness. Facial weakness is sometimes noted. Fasciculations are noted in about half of the patients (97) and are more common later in the disease course. Fasciculations in the limb muscles and thoracic wall muscles are com­mon. Calf pseudohypertrophy has been occasionally noted, but wasting of affected musculature is more prominent. Deep tendon reflexes are diminished and often become absent over time. Contractures are gen­erally mild as long as patients remain ambulatory. Scoliosis may be observed in SMA III, but it occurs less frequently and is less severe than scoliosis and SMA II. While no survival data exist for patients with SMA III, cases have been followed into the eighth decade with­out mechanical ventilation (57,98). Ventilatory fail­ure due to neuromuscular restrictive lung disease is a rare event in SMA III, occurring only in adulthood (57,117).

Zerres and Rudnik-Schoneborn (98) have proposed further subtypes, including SMA IIIa (walks without support; age of onset less than 3 years) and SMA IIIb (walks without support; age of onset 3-30 years). In their series, only 44% of SMA “Illa” patients remained ambulatory 20 years after onset of weakness, whereas 89% of “IIIb” patients remained ambulatory after a similar 20-year duration.

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Source: Alexander M.A., Matthews D.J.. Pediatric Rehabilitation: Principles and Practice. 4 th. åd. — New York: Demos Medical Publishing,2010. — 540 ð.. 2010
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