Clinical trials in rare disease: challenges and opportunities.
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ABSTRACT:
SUBMITTER: Augustine EF
PROVIDER: S-EPMC3964003 | biostudies-literature | 2013 Sep
REPOSITORIES: biostudies-literature
Publications
Clinical trials in rare disease: challenges and opportunities.
Augustine Erika F EF Adams Heather R HR Mink Jonathan W JW
Journal of child neurology 20130901 9
The neuronal ceroid lipofuscinoses constitute one of many groups of rare childhood diseases for which disease-modifying treatments are nonexistent. Disease-specific barriers to therapeutic success include incomplete understanding of disease pathophysiology and limitations of treatments that cannot adequately cross the blood-brain barrier to access the central nervous system. Therapeutic development in the neuronal ceroid lipofuscinoses shares many challenges with other rare diseases, such as inc ...[more]
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