2
Orphan Designations
0 approved
0
FDA Approvals
0
Active Trials
142 recruiting
9
Rare Diseases
across 9 areas
0
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Syntex-Synergen Neuroscience is a company with 2 orphan drug designations across 9 rare diseases. gene therapy candidates in 2 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 |
|---|---|---|
| Madras motor neuron disease | Ciliary neurotrophic factor, recombinant human | Des.TrialAppr. |
| acquired motor neuron disease | Ciliary neurotrophic factor, recombinant human | Des.TrialAppr. |
| frontotemporal dementia with motor neuron disease | Ciliary neurotrophic factor, recombinant human | Des.TrialAppr. |
| hereditary motor neuron disease | Ciliary neurotrophic factor, recombinant human | Des.TrialAppr. |
| motor neuron disease with dementia and ophthalmoplegia | Ciliary neurotrophic factor, recombinant human | Des.TrialAppr. |
| primary lateral sclerosis, adult, 1 | Ciliary neurotrophic factor, recombinant human | Des.TrialAppr. |
| spinal muscular atrophy | Ciliary neurotrophic factor, recombinant human | Des.TrialAppr. |
| spinal muscular atrophy, type III | Ciliary neurotrophic factor, recombinant human | Des.TrialAppr. |
| spinal muscular atrophy, type IV | Ciliary neurotrophic factor, recombinant human | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
6
overlap in 2+ diseases
2/9
candidate diseases
0
avg importance: 0
0
affecting portfolio
0% of portfolio targets high unmet need diseases
6
overlap in 2+ diseases
2/9
candidate diseases
0
avg importance: 0
0
affecting portfolio