14
Orphan Designations
5
FDA Approvals
35
Rare Diseases
0
News Articles
| Disease | Drug(s) | Designation | Approved |
|---|---|---|---|
| GNE myopathy | N-Acetylmannosamine (ManNAc) | Orphan Designation | - |
| Loeys-Dietz syndrome 4 | Carnitor | Orphan Designation | - |
| Rett syndrome | acetyl-l-carnitine (ALC) | Orphan Designation | - |
| bronchial endocrine tumor | CarnitorLevocarnitine | Orphan Designation | - |
| carnitine palmitoyl transferase 1A deficiency | Carnitor | Orphan Designation | - |
| central neurocytoma | Cystaran | Orphan Designation | - |
| cerebrotendinous xanthomatosis | chenodeoxycholic acid | Orphan Designation | - |
| cystinosis | Cystaran | Orphan Designation | - |
| fragile X syndrome | acetyl-l-carnitine | Orphan Designation | - |
| fumaric aciduria | Lactobacillus acidophilus and Bifidobacterium animalis subsp. lactis | Orphan Designation | - |
| genetic central precocious puberty in female | Carnitor | Orphan Designation | - |
| genetic generalized epilepsy | Carnitor | Orphan Designation | - |
| hereditary inclusion-body myopathy | N-Acetylmannosamine (ManNAc) | Orphan Designation | - |
| juvenile nephropathic cystinosis | Cystaran | Orphan Designation | - |
| malignant glioma | Procarbazine HCl | Orphan Designation | - |
| medium chain acyl-CoA dehydrogenase deficiency | levocarnitineCarnitor | Orphan Designation | - |
| metaphyseal dysplasia without hypotrichosis | CarnitorLevocarnitine | Orphan Designation | - |
| necrotizing enterocolitis | Lactobacillus acidophilus and Bifidobacterium animalis subsp. lactis | Orphan Designation | - |
| nephropathic cystinosis | Cystaran | Orphan Designation | - |
| ocular cystinosis | Cystaran | Orphan Designation | - |
| pediatric hepatocellular carcinoma | Levocarnitine | Orphan Designation | - |
| pediatric ovarian dysgerminoma | Levocarnitine | Orphan Designation | - |
| pediatric-onset Graves disease | Levocarnitine | Orphan Designation | - |
| perinatal necrotizing enterocolitis | Lactobacillus acidophilus and Bifidobacterium animalis subsp. lactis | Orphan Designation | - |
| primary biliary cholangitis | Carnitor | Orphan Designation | - |
| primary cutaneous lymphoma | Carnitor | Orphan Designation | - |
| primary hyperoxaluria | Carnitor | Orphan Designation | - |
| renal tubular dysgenesis of genetic origin | Carnitor | Orphan Designation | - |
| secondary central precocious puberty in female | levocarnitineCarnitor | Orphan Designation | - |
| secondary hyperparathyroidism of renal origin | Carnitor | Orphan Designation | - |
| secondary hypertrophic osteoarthropathy | levocarnitineCarnitor | Orphan Designation | - |
| secondary vasculitis | Carnitor | Orphan Designation | - |
| skin disorder | CarnitorLevocarnitine | Orphan Designation | - |
| systemic primary carnitine deficiency disease | Carnitor | Orphan Designation | - |
| thrombocytopenia 12 with or without myopathy | N-Acetylmannosamine (ManNAc) | Orphan Designation | - |