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Table 2 Major neurological and MRI diffusion changes, by subject, for the phase I trial in Pelizaeus-Merzbacher disease

From: Clinical translation of human neural stem cells

Subject

Neurological and radiological changes

Subject 1 (16 m of age at transplant)

Tracheostomy and gastrostomy at baseline

 

Remained neurologically stable, but was noted to have reduced nightly CPAP at 12 months

 

Increased FA by MRI

Subject 2 (42 m of age at transplant)

Developed improved truncal support and the ability to take steps with assistance. He also began speaking audible single words and the ability to follow two-step commands

 

Increased FA by MRI

Subject 3 (14 m of age at transplant)

Tracheostomy and gastrostomy at baseline

 

Developed upper extremity antigravity strength and to take some solid foods by mouth

 

Nightly CPAP dependency reduced

 

Greatest increase in FA by MRI; but comparable to ‘control’ regions

Subject 4 (66 m of age at transplant)

Developed improved truncal support and progressed from the use of a walker with significant support at baseline to walking with minimal assistance

 

Developed the ability for self-feeding and to follow two-step commands

 

Increased FA by MRI

  1. CPAP, continuous positive airway pressure; FA, fractional anisotropy; m, months.