Disorder
|
Number Treated
|
Outcome
|
Ref.
|
|
Hurler's syndrome
|
20
|
17 of the 20 children were alive a median of 905 days after transplantation, with complete donor chimerism and normal peripheral-blood alpha-L-iduronidase activity
|
[100]
|
Duchenne muscular dystrophy
|
1
|
On 42nd day, physical examination revealed obviously improvement in walking, turning the body over, and standing up
|
[101]
|
Malignant infantile osteopetrosis
|
1
|
Normalization of spine bone mineral density.
|
[102]
|
Rothmund-Thomson syndrome
|
1
|
Complete immune reconstitution
|
[55]
|
Buerger's disease
|
4
|
Ischemic rest pain suddenly disappeared. Digital capillaries were increased in number and size.
|
[84]
|
Spinal Cord Injury
|
1
|
Improved sensory perception and movement in the SPI patient's hips and thighs within 41 days of cell transplantation. Regeneration of the spinal cord at the injured site
|
[85]
|
Krabbe's disease
|
25
|
Progressive central myelination and continued gains in developmental skills, and most had age-appropriate cognitive function and receptive language skills in patient subset
|
[14]
|
Omenn syndrome
|
1
|
T cell reconstitution
|
[103]
|
Non-healing wounds
|
2
|
Accelerated healing
|
[86]
|
Refractory anemia
|
3
|
All patients are alive and free of disease at between 17 and 39 months after cord blood administration
|
[104]
|
Diamond-Blackfan anemia
|
1
|
Successful seroconversion to vaccines (diphtheria, pertussis, tetanus, rubella, measles, and BCG) administered 22–34 months post-transplant.
|
[105]
|
Severe chronic active Epstein-Barr virus
|
1
|
Complete remission without circulating EBV-DNA has continued for 15 months transplant.
|
[106]
|
Behcet's disease
|
1
|
Twenty-three months after CBT, the patient is doing well and has no signs or symptoms of Behcet's disease
|
[9]
|
Mucopolysaccharidosis type IIB (Hunter syndrome)
|
1
|
Two years after transplant approximately 55% normal plasma iduronate sulfatase. activity has been restored and abnormal urinary excretion of glycosaminoglycans has nearly completely resolved.
|
[107]
|
Answers to all your Biology Questions
