1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
3
Rare Diseases
across 6 areas
0
News (30d)
Quiet
Kurt+Peter Foundation is a company with 1 orphan drug designation across 3 rare diseases.
Disease-by-disease pipeline progression from orphan designation through clinical trials to FDA approval.
Source: FDA Orphan Drug Designations, ClinicalTrials.gov, Drugs@FDA
| Disease | Drug(s) | Stage |
|---|---|---|
| autosomal recessive limb-girdle muscular dystrophy type 2B | One, two, three, or four antisense oligonucleotides of Phosphorodiamidate morpholino oligomer combination that skips exons 4, 5, 6, and 7 of the gamma sarcoglycan (SGCG) gene | Des.TrialAppr. |
| autosomal recessive limb-girdle muscular dystrophy type 2C | One, two, three, or four antisense oligonucleotides of Phosphorodiamidate morpholino oligomer combination that skips exons 4, 5, 6, and 7 of the gamma sarcoglycan (SGCG) gene | Des.TrialAppr. |
| autosomal recessive limb-girdle muscular dystrophy type 2I | One, two, three, or four antisense oligonucleotides of Phosphorodiamidate morpholino oligomer combination that skips exons 4, 5, 6, and 7 of the gamma sarcoglycan (SGCG) gene | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
1
overlap in 2+ diseases
0/3
candidate diseases
0
avg importance: 0
0
affecting portfolio