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Table 7 Change from baseline in total grey matter volume at week 52

From: Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial

patient

age

Total grey matter volume (mm3)

BL

26 wk

52 wk

52wk-BL

202

9.0

797,018

792,018

788,648

-8,370

203

6.1

747,735

768,848

nd

+21,113a

204

14.8

517,491

604,125

616,132

+98,641b

205

5.0

710,691

759,444

749,177

+38,486

206

3.1

621,494

673,817

703,973

+82,478

207

5.9

607,678

590,810

598,460

-9,218

211

2.5

589,439

npc

npc

 

213

15.7

874,710

885,006

863,862

-10,848

214

2.0

744,555

764,571

780,779

+36,224

215

12.1

668,638

663,441

676,069

+7,431

216

7.8

739,492

708,228

717,836

-21,656

mean

 

692,631

  

+15,071

SEM

 

34,396

  

11,052

  1. BL baseline, nd volumetrics analysis could not be performed
  2. a52-week volumetrics not available; difference computed from 26-week volume
  3. bPatient with braces; volumes not used to compute mean difference at 52 weeks
  4. cnp=not performed; volumetric analysis could not be completed
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Orphanet Journal of Rare Diseases

ISSN: 1750-1172