May 02 2013
A groundbreaking clinical trial of gene therapy for a form of congenital blindness, sponsored by The Children’s Hospital of Philadelphia in collaboration with Penn Medicine, was recently recognized with the Distinguished Clinical Research Achievement Award from the Clinical Research Forum, an organization of clinical research centers, industry, and volunteer groups.
Permanent link to this article: http://www.research.chop.edu/blog/chop-penn-gene-therapy-trial-for-blindness-honored/
Dec 28 2012
Canavan disease is a rare inherited neurological disorder with devastating effect. The lack of a specific enzyme, called aspartoacylase, causes the body’s central nervous system to break down. The disease is usually fatal before a child reaches the teenage years.
Permanent link to this article: http://www.research.chop.edu/blog/gene-therapy-shows-promise-for-helping-children-with-canavan-disease/
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