Loading disease data
| Disease | Prevalence | Drugs | Proteins | |
|---|---|---|---|---|
| Obesity (OBESITY) | Uncommon | 286 | 2 | |
| Type 2 diabetes mellitus (T2D) | Common | 377 | 2 | |
| Abdominal obesity-metabolic syndrome 3 (AOMS3) | Common | — | 1 | |
| Adenylosuccinase deficiency (ADSLD) | Ultra-Rare | 1 | 1 | |
| Adrenoleukodystrophy, pseudoneonatal (Pseudo-NALD) | Ultra-Rare | — | 1 | |
| Alpha-1-antitrypsin deficiency (A1ATD) | Uncommon | 4 | 1 | |
| Amyloidosis, hereditary systemic 1 (AMYLD1) | Uncommon | — | 1 | |
| Amyloidosis, hereditary systemic 6 (AMYLD6) | N/A | — | 1 | |
| Argininemia (ARGIN) | Ultra-Rare | — | 1 | |
| Argininosuccinic aciduria (ARGINSA) | Rare | — | 1 | |
| Aromatic L-amino-acid decarboxylase deficiency (AADCD) | Ultra-Rare | — | 1 | |
| Aspartylglucosaminuria (AGU) | Rare | — | 1 | |
| Biotinidase deficiency (BTD deficiency) | Rare | — | 1 | |
| Butyrylcholinesterase deficiency (BCHED) | Rare | 1 | 1 | |
| Ceroid lipofuscinosis, neuronal, 10 (CLN10) | Ultra-Rare | — | 1 | |
| Ceroid lipofuscinosis, neuronal, 11 (CLN11) | Ultra-Rare | — | 1 | |
| Ceroid lipofuscinosis, neuronal, 2 (CLN2) | Ultra-Rare | 1 | 1 | |
| Combined oxidative phosphorylation deficiency 16 (COXPD16) | Ultra-Rare | — | 1 | |
| Combined saposin deficiency (PSAPD) | Ultra-Rare | — | 1 | |
| Congenital disorder of glycosylation 1A (CDG1A) | Rare | — | 1 | |
| Developmental and epileptic encephalopathy 29 (DEE29) | N/A | — | 1 | |
| Dihydropyrimidinase deficiency (DPYSD) | Ultra-Rare | — | 1 | |
| Disordered steroidogenesis due to cytochrome P450 oxidoreductase deficiency (DISPORD) | Rare | — | 1 | |
| Dystonia, DOPA-responsive, due to sepiapterin reductase deficiency (DRDSPRD) | Ultra-Rare | — | 1 | |
| Fabry disease (FD) | Uncommon | 16 | 1 | |
| Fanconi anemia complementation group L (FANCL) | Rare | — | 1 | |
| Fish-eye disease (FED) | Ultra-Rare | 19 | 1 | |
| Fructosuria (FRUCT) | N/A | — | 1 | |
| Galactosemia 2 (GALAC2) | Rare | — | 1 | |
| Galactosemia 3 (GALAC3) | Rare | — | 1 | |
| Galactosialidosis (GSL) | Very Rare | — | 1 | |
| Gaucher disease, atypical, due to saposin C deficiency (GDSAPC) | Rare | — | 1 | |
| Glutamate formiminotransferase deficiency (FIGLU-URIA) | N/A | — | 1 | |
| Glutaric aciduria 1 (GA1) | Common | — | 1 | |
| Glycine N-methyltransferase deficiency (GNMT deficiency) | N/A | — | 1 | |
| Glycogen storage disease 13 (GSD13) | Ultra-Rare | — | 1 | |
| Glycogen storage disease 9C (GSD9C) | Rare | — | 1 | |
| Glycogen storage disease of heart lethal congenital (GSDH) | Ultra-Rare | — | 1 | |
| GM2-gangliosidosis 1 (GM2G1) | Very Rare | 8 | 1 | |
| GM2-gangliosidosis AB (GM2GAB) | Ultra-Rare | — | 1 | |
| Haim-Munk syndrome (HMS) | Ultra-Rare | — | 1 | |
| Heme oxygenase 1 deficiency (HMOX1D) | Ultra-Rare | — | 1 | |
| Hemochromatosis 5 (HFE5) | Ultra-Rare | — | 1 | |
| HSD10 mitochondrial disease (HSD10MD) | Ultra-Rare | — | 1 | |
| Hypercholesterolemia, familial, 3 (FHCL3) | Very Rare | — | 1 | |
| Hyperimmunoglobulinemia D and periodic fever syndrome (HIDS) | Ultra-Rare | — | 1 | |
| Hyperphenylalaninemia, BH4-deficient, A (HPABH4A) | Very Rare | — | 1 | |
| Hypertryptophanemia (HYPTRP) | Ultra-Rare | — | 1 | |
| Hypoalphalipoproteinemia, primary, 2 (FHA2) | Ultra-Rare | — | 1 | |
| Hypoalphalipoproteinemia, primary, 2, intermediate (FHA2I) | N/A | — | 1 |