Therapeutic Peptides intermediate
Peptide Rare Disease Therapeutics
Therapeutic peptides for rare diseases including enzyme replacement therapies and hormone replacement.
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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