1
Orphan Designations
0 approved, 1 designated
0
FDA Approvals
0
Active Trials
1
Rare Diseases
across 3 areas
1
News (30d)
Muscular Dystrophy Association, Inc. is a company with 1 orphan drug designation across 1 rare disease. 1 news article in the past 30 days.
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 |
|---|---|---|
| congenital myasthenic syndrome | adeno-associated virus serotype 9 human choline acetyltransferase (AVCHAT-01X) | Des.TrialAppr. |
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/1
candidate diseases
1
avg importance: 40
0
affecting portfolio
0% of portfolio targets high unmet need diseases
0
overlap in 2+ diseases
0/1
candidate diseases
1
avg importance: 40
0
affecting portfolio