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Pediatr Clin North Am. 2015 Jun;62(3):649-66. doi: 10.1016/j.pcl.2015.03.006. Epub 2015 Apr 8.

Emerging treatments for pediatric leukodystrophies.

Author information

1
Department of Neurology, Children's National Health System, 111 Michigan Avenue, Northwest, Washington, DC 20010, USA; Center for Genetic Medicine Research, Children's National Health System, 111 Michigan Avenue, Northwest, Washington, DC 20010, USA.
2
Department of Neurology, Lucile Packard Children's Hospital, Stanford University School of Medicine, 730 Welch Rd, Palo Alto, CA 94304, USA.
3
Department of Integrated Systems Biology, George Washington University School of Medicine, 2150 Pennsylvania Ave NW, Washington, DC 20037, USA.
4
Department of Neurology, Children's National Health System, 111 Michigan Avenue, Northwest, Washington, DC 20010, USA; Center for Genetic Medicine Research, Children's National Health System, 111 Michigan Avenue, Northwest, Washington, DC 20010, USA; Department of Integrated Systems Biology, George Washington University School of Medicine, 2150 Pennsylvania Ave NW, Washington, DC 20037, USA. Electronic address: avanderv@childrensnational.org.

Abstract

The leukodystrophies are a heterogeneous group of inherited disorders with broad clinical manifestations and variable pathologic mechanisms. Improved diagnostic methods have allowed identification of the underlying cause of these diseases, facilitating identification of their pathologic mechanisms. Clinicians are now able to prioritize treatment strategies and advance research in therapies for specific disorders. Although only a few of these disorders have well-established treatments or therapies, a number are on the verge of clinical trials. As investigators are able to shift care from symptomatic management of disorders to targeted therapeutics, the unmet therapeutic needs could be reduced for these patients.

KEYWORDS:

Disease modifying; Gene therapy; Genomics; Leukodystrophy; Stem cell; Symptomatic; Therapy

PMID:
26022168
PMCID:
PMC5712822
DOI:
10.1016/j.pcl.2015.03.006
[Indexed for MEDLINE]
Free PMC Article

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