A Sandhoff disease that occurs in an adult.
Comprehensive, easy-to-understand information about this condition
How we create this content →Helpful links for rare disease information and support
Questions that may be helpful when speaking with your healthcare team
Helpful links for rare disease information and support
Questions that may be helpful when speaking with your healthcare team
Helpful links for rare disease information and support
Questions that may be helpful when speaking with your healthcare team
Clinical profile data for this condition is not yet available. Phenotype information may still be loading below.
European rare disease database
Genetic and Rare Diseases Info Center
AI-Generated Content: This summary was generated using AI. Always consult with qualified healthcare providers for medical guidance.
Kisho delivers this disease record via API, including phenotypes (HPO), genes, orphan drug designations, screening status, and PAG mapping, with version history and governance.
Organizations with orphan designations or approved therapies for this disease
Ablynx, a wholly owned subsidiary of Sanofi Group
Other
Alexion Pharmaceuticals Inc.
Other
Apellis Pharmaceuticals, Inc.
Other
AstraZeneca Pharmaceuticals LP
Other
Chimerix, Inc.
Other
EMD Serono Research and Development Institute, Inc.
Other
Epizyme, Inc.
Other
Genentech, Inc.
Other
GlaxoSmithKline LLC
Other
Novartis Pharmaceuticals Corporation
Other
Pfizer, Inc.
Other
Pharmacyclics LLC
Other
Regeneron Pharmaceuticals, Inc.
Other
Rh Pharmaceuticals, Inc.
Other
Servier Pharmaceuticals LLC
Other
SpringWorks Therapeutics, Inc.
Other
Swedish Orphan Biovitrum AB
Other