3
Orphan Designations
0 approved, 3 designated
0
FDA Approvals
0
Active Trials
6
Rare Diseases
across 9 areas
0
News (30d)
Quiet
Cure Rare Disease is a company with 3 orphan drug designations across 6 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 dominant cerebellar ataxia | a single stranded 2-methoxyethyl RNA nucleotide with a full-length phosphorothioate backbone | Des.TrialAppr. |
| autosomal recessive limb-girdle muscular dystrophy | adeno associated vector (serotype AAV MYO2) encoding the fukutin related protein (ssAAVMYO2.tMCK.FKRP) | Des.TrialAppr. |
| congenital muscular dystrophy | adeno associated vector (serotype AAV MYO2) encoding the fukutin related protein (ssAAVMYO2.tMCK.FKRP) | Des.TrialAppr. |
| limb-girdle muscular dystrophy | adeno associated vector (serotype AAV MYO2) encoding the fukutin related protein (ssAAVMYO2.tMCK.FKRP) | Des.TrialAppr. |
| spinocerebellar ataxia 43 | a single stranded 2-methoxyethyl RNA nucleotide with a full-length phosphorothioate backbone | Des.TrialAppr. |
| spinocerebellar ataxia 7 | a single stranded 2-methoxyethyl RNA nucleotide with a full-length phosphorothioate backbone | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
3
overlap in 2+ diseases
0/6
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
3
overlap in 2+ diseases
0/6
candidate diseases
0
avg importance: 0
0
affecting portfolio