Leukotriene C4 synthase deficiency is an extremely rare fatal neurometabolic developmental disorder characterized clinically by muscular hypotonia, psychomotor retardation, failure to thrive, and micr...
Comprehensive, easy-to-understand information about this condition
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Organizations with orphan designations or approved therapies for this disease
AGI Dermatics
Other
Aldagen, Inc.
Other
Angion Biomedica Corporation
Other
Ferring Pharmaceuticals Inc.
Other
GE Healthcare Inc.
Other
Genentech, Inc.
Other
Invero Pharma, Inc.
Other
Janssen Research & Development, LLC
Other
Knoll Pharmaceutical Company
Other
Mallinckrodt Pharmaceuticals Ireland Limited
Other
QOL Medical, LLC
Other
Questcor Pharmaceuticals, Inc.
Other
Stealth Biotherapeutics Inc.
Other
Teijin America, Inc.
Other