Accepted for Publication: July 7, 2013.
Published Online: October 23, 2013. doi:10.1001/jamadermatol.2013.6683.
Study concept and design: Laugel, Dollfus, Lipsker.
Acquisition of data: Frouin, Laugel, Durand, Lipsker.
Analysis and interpretation of data: Frouin, Laugel, Lipsker.
Drafting of the manuscript: Frouin, Laugel, Durand, Lipsker.
Critical revision of the manuscript for important intellectual content: Laugel, Dollfus.
Obtained funding: Laugel, Dollfus.
Administrative, technical, and material support: Laugel, Durand.
Study supervision: Laugel, Lipsker.
Conflict of Interest Disclosures: None reported.
Funding/Support: This work was supported by grants from the French Ministry of Health (National Program for Clinical Research) and from the Institut des Maladies Rares.
Role of the Sponsor: The French Ministry of Health and the Institut des Maladies Rares had no role in the design and conduct of the study; collection, management, analysis, and interpretation of the data; preparation, review, or approval of the manuscript; and decision to submit the manuscript for publication.
Additional Contributions: We thank all patients and families who collaborated in this work, the support groups Amy and Friends and Les P’tits Bouts, and all physicians in charge of the care for these patients who helped to collect relevant clinical information. No financial compensation was given for these services.