Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
This study is currently recruiting participants.
Verified March 2014 by Duke University
Rare Diseases Clinical Research Network
Information provided by (Responsible Party):
First received: August 13, 2012
Last updated: March 12, 2014
Last verified: March 2014
No Study Results Posted on ClinicalTrials.gov for this Study
|Study Status:||This study is currently recruiting participants.|
|Estimated Study Completion Date:||August 2019|
|Estimated Primary Completion Date:||August 2019 (Final data collection date for primary outcome measure)|
Banugaria SG, Prater SN, Ng YK, Kobori JA, Finkel RS, Ladda RL, Chen YT, Rosenberg AS, Kishnani PS. The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease. Genet Med. 2011 Aug;13(8):729-36. doi: 10.1097/GIM.0b013e3182174703.
Messinger YH, Mendelsohn NJ, Rhead W, Dimmock D, Hershkovitz E, Champion M, Jones SA, Olson R, White A, Wells C, Bali D, Case LE, Young SP, Rosenberg AS, Kishnani PS. Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease. Genet Med. 2012 Jan;14(1):135-42. doi: 10.1038/gim.2011.4.