Rare Disease Report
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Patients & Caregivers

Leukodystrophy in Pompe Disease

FEBRUARY 23, 2017
Alexander Bloomfield, MBBS


Alexander Bloomfield, MBBS, MSc of the Royal Manchester Children's Hospital discusses a patient with infantile Pompe disease who has leukodystrophy. This is a potentially important consideration for infantile Pompe patients with sudden onset of gait abnormalities.

Reference

Bloomfield A, Fletcher J, Hensman P, et al. Rapid progressive leukodystrophy in early childhood: a new phenotype for treated infantile onset Pompe patients? Presented at 13th Annual WORLDSymposium; San Diego, CA; February 13-16, 2017.


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