Therapeutic Peptides intermediate
Peptide Rare Disease Therapeutics
Therapeutic peptides for rare diseases including enzyme replacement therapies and hormone replacement. This peptide or oligopeptide is studied for its biolog...
By Encyclopeptide Editorial | 1 min read
rare-disease enzyme-replacement genetic therapeutic orphan-drug
Overview
Rare diseases often involve deficiency of specific enzymes or hormones, making them ideal targets for peptide-based replacement therapies.
Major Rare Disease Peptides
| Drug | Indication | Mechanism |
|---|---|---|
| Agalsidase beta | Fabry disease | Enzyme replacement |
| Imiglucerase | Gaucher disease | Enzyme replacement |
| Laronidase | MPS I | Enzyme replacement |
Challenges
- High cost ($100,000-$500,000/year)
- IV administration requirements
- Immunogenicity
- Access and equity
Chemical Identity
| Property | Value |
|---|---|
| Name | Peptide Rare Disease Therapeutics |
| Category | Therapeutic Peptides |
References
- Encyclopeptide Database. “Peptide Rare Disease Therapeutics” monograph. encyclopeptide.com.
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