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Disease Prioritization Data Package
This package has 16 modules. The Rare Diseases Module has 5 tables. Preview the first five rows of each table or Explore the schema
Rare Diseases Module
5 Tables
Describes the status of a drug intended for use against rare diseases. The dataset offers a view of the orphan designations without being specifically tied to any one set of regulatory standards or rules.
id | trial_id | biodb_id | label |
|---|---|---|---|
3 | NCT02836340 | BE0004550 | protein |
8 | NCT02836340 | BE0001177 | protein |
13 | NCT04143295 | BE0000178 | protein |
18 | NCT04143295 | BE0000178 | gene |
23 | NCT01337986 | BE0009847 | gene |
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id | mention_id | string | section |
|---|---|---|---|
3 | 3 | NSE | description |
8 | 8 | s100b | description |
13 | 13 | MCT8 | description |
18 | 13 | MCT8 | title |
23 | 13 | MCT8 | official_title |
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id | condition_id | source | designation_status | designation_date |
|---|---|---|---|---|
2 | 7749 | FDA | designated_withdrawn | 2015-05-04 |
7 | 157373 | FDA | designated | 2017-07-03 |
12 | 550 | FDA | designated | 2007-12-18 |
17 | 38696 | FDA | designated | 1996-10-29 |
22 | 71340 | FDA | designated_withdrawn | 1993-11-22 |
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id | drug_id | orphan_designation_id |
|---|---|---|
2 | 12476 | 2 |
7 | 27573 | 7 |
12 | 27578 | 12 |
17 | 1050 | 17 |
22 | 27598 | 22 |
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cf_grid_key | orphan_designation_id | generic_name | orphan_designation | designation_date | designation_status | marketing_approval_date | exclusivity_end_date | approved_labeled_indication | exclusivity_protected_indication |
|---|---|---|---|---|---|---|---|---|---|
000183 | 22170 | physostigmine salicylatephysostigmine salicylate | Treatment of Friedreich's and other inherited ataxias. | 1985-01-16 | designated_withdrawn | ||||
000383 | 29221 | Diaziquone | Treatment of primary brain malignancies (grade III and IV astrocytomas). | 1983-11-10 | designated_withdrawn | ||||
000583 | 28700 | Selegiline HCl | As an adjuvant to levodopa and carbidopa treatment of idiopathic Parkinson's disease (paralysis agitans), postencephalitic Parkinsonism, and symptomatic Parkinsonism. | 1984-11-07 | designated_approved | 1989-06-05 | 1996-06-05 | ||
000683 | 25260 | Hemin | Amelioration of recurrent attacks of acute intermittent porphyria (AIP) temporarily related to the menstrual cycle in susceptible women and similar symptoms which occur in other patients with AIP, porphyria variegata and hereditary coproporphyria. | 1984-03-16 | designated_approved | 1983-07-20 | 1990-07-20 | ||
000883 | 10492 | Teniposide | Treatment of refractory childhood acute lymphocytic leukemia. | 1984-11-01 | designated_approved | 1992-07-14 | 1999-07-14 | Induction therapy in patients with refractory childhood acute lymphoblastic leukemia when used in combination with other approved anticancer agents. |
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Showing 5 of 5 tables