Abstract
A large body of experience has been compiled in different countries, documenting the
efficacy of adenocorticotropic hormone (ACTH) for infantile spasms. This is important,
because it may serve as a key for understanding this disorder, as well as for designing
better medicines. However, significant discrepancies exist among studies originating
in different countries regarding the relative efficacy of small or large ACTH doses.
These differences may be caused by a number of factors, including potential genetic
or environmental-related differences in the biology of the disorder or associated
genetic components that determine responsiveness to ACTH. In addition, striking differences
in the preparations used around the world may be responsible. These include bio-availability
and extent of blood brain barrier penetration, efficacy in activating the efficacy-mediating
‘ACTH receptors’, the presence in certain preparations of competing analogs, and others.
These issues should not detract from the overall agreement that ACTH might be the
most useful medication currently available to treat WS.
Keywords
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Article info
Publication history
Accepted:
August 21,
2001
Received in revised form:
July 12,
2001
Received:
June 11,
2001
Identification
Copyright
© 2001 Published by Elsevier Inc.