Browse the GARD list of rare diseases and related terms to find topics of interest to you. This list includes the main name for each condition, as well as alternate names. Inclusion on this list does not serve as official recognition by the NIH that a disease is rare. Some conditions that are not considered rare are on this list and are labeled accordingly.Read more about which diseases are included on the GARD website.
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- VACTERL association
- VACTERL association with hydrocephaly - See VACTERL hydrocephaly
- VACTERL association with hydrocephaly, X-linked
- VACTERL hydrocephaly
- Vacuolar cardiomyopathy and myopathy X-linked - See Danon disease
- Vacuolating megalencephalic leukoencephalopathy with subcortical cysts - See Megalencephalic leukoencephalopathy with subcortical cysts
- Vagal paraganglioma - See Glomus vagale tumor
- Vagina, absence of
- Vaginal cancer
- Vagneur Triolle Ripert syndrome
- Valine metabolic defect - See HIBCH deficiency
- Valine transaminase deficiency - See Valinemia
- Valinemia
- Valley fever - See Coccidioidomycosis
- Valproic acid embryopathy - See Fetal valproate syndrome
- Valvar pulmonary stenosis - See Pulmonary valve stenosis
- Valvate pulmonary stenosis - See Pulmonary valve stenosis
- Valvular aortic stenosis - See Aortic valve stenosis
- Valvular heart disease, congenital - See Cardiac valvular dysplasia, X-linked
- Valvular pulmonary stenosis - See Pulmonary valve stenosis
- Valvular pulmonic stenosis - See Pulmonic stenosis
- Van Allen Myhre syndrome
- VAN ASPEREN SYNDROME - See Chromosome 17q11.2 deletion syndrome
- Van Benthem-Driessen-Hanveld syndrome
- Van Bervliet syndrome - See Arthrogryposis IUGR thoracic dystrophy
- Van Biervliet Hendrickx Van Ertbruggen syndrome - See Craniofacial dysostosis arthrogryposis progeroid appearence
- Van Bogaert-Hozay syndrome
- Van Buchem disease - See Hyperostosis corticalis generalisata
- Van Buchem disease type 2
- Van De Berghe Dequeker syndrome - See Ulnar hypoplasia lobster claw deformity of feet
- Van Den Bosch syndrome
- Van Den Ende Brunner syndrome - See Ectrodactyly cardiopathy dysmorphism
- Van den Ende Gupta syndrome - See Marden Walker like syndrome
- Van der Hoeve Halbertsma Waardenburg Gualdi Syndrome - See Waardenburg syndrome
- Van der Hoeve syndrome - See Osteogenesis imperfecta type I
- Van der Woude syndrome
- Van der Woude syndrome 2
- Van Goethem syndrome - See Radio-digito-facial dysplasia
- Van Lohuizen syndrome - See Cutis marmorata telangiectatica congenita
- Van Maldergem syndrome - See Cerebro-facio-articular syndrome
- Van Maldergem Wetzburger Verloes syndrome - See Cerebro-facio-articular syndrome
- Van Regemorter Pierquin Vamos syndrome
- Vanishing bone disease - See Gorham's disease
- Vanishing white matter disease - See Leukoencephalopathy with vanishing white matter
- Vanishing white matter leukodystrophy - See Leukoencephalopathy with vanishing white matter
- Váradi syndrome - See Orofaciodigital syndrome 6
- Váradi-Papp syndrome - See Orofaciodigital syndrome 6
- Variant A or HHV-6A - See HHV-6 encephalitis
- Variant angina - See Prinzmetal's variant angina
- Variant B or HHV-6B - See HHV-6 encephalitis
- Variant CJD - See Variant Creutzfeldt-Jakob disease
- Variant Creutzfeldt-Jacob disease - See Variant Creutzfeldt-Jakob disease
- Variant Creutzfeldt-Jakob disease
- Variant of Mucha-Habermann disease - See Febrile Ulceronecrotic Mucha-Habermann disease
- Variant phenylketonuria - See Mild phenylketonuria
- Variant PKU - See Mild phenylketonuria
- Varicella Embryopathy - See Congenital varicella syndrome
- Varicella virus antenatal infection - See Congenital varicella syndrome
- Variegate porphyria
- Variola - See Smallpox
- Varioliform gastritis - See Chronic erosive gastritis
- Vascular compression of the duodenum - See Superior mesenteric artery syndrome
- Vascular EDS - See Vascular Ehlers-Danlos syndrome
- Vascular Ehlers-Danlos syndrome
- Vascular hyalinosis
- Vascular purpura - See Henoch-Schonlein purpura
- Vasculitis - not a rare disease
- Vasculitis due to ADA2 deficiency - See Adenosine Deaminase 2 deficiency
- Vasculitis due to DADA2 - See Adenosine Deaminase 2 deficiency
- Vasculitis lymphocytic, nodular - See Lymphocytic vasculitis
- Vasculitis, autoimmune - See Vasculitis - not a rare disease
- Vasoactive intestinal peptide (VIP) tumor - See VIPoma
- Vasoactive intestinal peptide-producing tumor - See VIPoma
- Vasopressin-resistant diabetes insipidus - See Nephrogenic diabetes insipidus
- Vasospastic angina - See Prinzmetal's variant angina
- Vasquez Hurst Sotos syndrome
- VATER association - See VACTERL association
- VBCH - See Hyperostosis corticalis generalisata
- VBCH2 - See Van Buchem disease type 2
- VCFS - See 22q11.2 deletion syndrome
- VCJD - See Variant Creutzfeldt-Jakob disease
- VDEGS - See Marden Walker like syndrome
- VDWS - See Van der Woude syndrome
- vEDS - See Vascular Ehlers-Danlos syndrome
- Vein of Galen aneurysm
- Vein of Galen aneurysm malformation - See Vein of Galen aneurysm
- Velocardiofacial syndrome - See 22q11.2 deletion syndrome
- Velofacioskeletal syndrome
- Velopharyngeal incompetence - See Palatopharyngeal incompetence
- Velopharyngeal insufficiency - See Palatopharyngeal incompetence
- Venezuelan equine encephalitis
- Ventricular extrasystoles perodactyly Robin sequence - See Ventricular extrasystoles with syncopal episodes - perodactyly - Robin sequence
- Ventricular extrasystoles with syncopal episodes - perodactyly - Robin sequence
- Ventricular extrasystoles with syncope, perodactyly, and Robin sequence - See Ventricular extrasystoles with syncopal episodes - perodactyly - Robin sequence
- Ventricular familial preexcitation syndrome - See Wolff-Parkinson-White syndrome - not a rare disease
- Ventricular fibrillation with prolonged QT interval - See Long QT syndrome 1
- Ventricular fibrillation, idiopathic
- Ventricular fibrillation, paroxysmal familial - See Paroxysmal ventricular fibrillation
- Ventricular septal defects
- Ventriculo-arterial discordance, isolated
- Ventriculomegaly - See Congenital hydrocephalus
- Ventriculo-radial syndrome - See Holt-Oram syndrome
- Ventruto Digirolamo Festa syndrome
- Verheij syndrome - See Chromosome 8q24.3 deletion syndrome
- Verloes Bourguignon syndrome
- Verloes Gillerot Fryns syndrome - See Male pseudohermaphroditism intellectual disability syndrome, Verloes type
- Verloes syndrome - See Male pseudohermaphroditism intellectual disability syndrome, Verloes type
- Verloes Van Maldergem Marneffe syndrome
- Verloes-David syndrome - See Mesomelia-synostoses syndrome
- Verloes-Gillerot-Fryns syndrome - See Male pseudohermaphroditism intellectual disability syndrome, Verloes type
- Verloove Vanhorick Brubakk syndrome
- Verma Naumoff syndrome - See Short rib-polydactyly syndrome type 3
- Vermiculate atrophoderma, milia, hypotrichosis, trichoepitheliomas, basal cell carcinomas and peripheral vasodilation with cyanosis - See Rombo syndrome
- Vernal keratitis
- Vernal keratoconjunctivitis
- Verner-Morrison syndrome - See WDHA syndrome
- Verrucous epidermal nevus - See Inflammatory linear verrucous epidermal nevus
- Verrucous nevus - See Inflammatory linear verrucous epidermal nevus
- Verrucous nevus acanthokeratolytic
- Vertebral (V), anal (A), cardiac (C), tracheoesophageal (TE), renal (R) and limb (L) anomalies and hydrocephaly - SeeVACTERL hydrocephaly
- Vertebral artery syndrome - See Wallenberg syndrome
- Vertebral body fusion overgrowth
- Vertebral fusion posterior lumbosacral blepharoptosis
- Vertebral fusion with carpal coalition - See Spondylocarpotarsal synostosis syndrome
- Vertebrobasilar migraine - See Migraine with brainstem aura
- Vertical talus - See Congenital vertical talus
- Vertical talus, congenital - See Congenital vertical talus
- Very long-chain acyl-CoA dehydrogenase deficiency - See VLCAD deficiency
- Vesicles, celosomia, short neck, microcornea and dextrocardia - See Van Allen Myhre syndrome
- Vesicoenteric fistula - See Enterovesical fistula
- Vessel’s syndrome - See Proximal symphalangism
- Vestibular schwannoma - See Acoustic neuroma
- Vestibulocerebellar disorder with predominant ocular signs - See Nystagmus 4, congenital, autosomal dominant
- Vestibulocochlear dysfunction, progressive
- VGAM - See Vein of Galen aneurysm
- VHL - See Von Hippel-Lindau disease
- VHL syndrome - See Von Hippel-Lindau disease
- VI nerve palsy - See Sixth nerve palsy
- Vibratory angioedema - See Vibratory urticaria
- Vibratory urticaria
- Vibrio cholerae infection - See Cholera
- Vibrio vulnificus infection
- Vici syndrome
- Viljoen Kallis Voges syndrome
- Viljoen Smart syndrome - See Microphthalmia syndromic 8
- Viljoen Winship syndrome
- Vincent's gingivitis - See Acute necrotizing ulcerative gingivitis
- Vincent's infection - See Acute necrotizing ulcerative gingivitis
- Vincent's stomatitis - See Acute necrotizing ulcerative gingivitis
- VIPoma
- Vipoma Syndrome - See WDHA syndrome
- VIP-secreting tumor - See VIPoma
- Viral hemorrhagic fever
- Viral hepatitis non-A,-B,-C,-D,-E - See Non-A-E hepatitis
- Virilizing ovarian tumor
- Virus associated hemophagocytic syndrome
- Visceral heterotaxy - See Heterotaxy
- Visceral leishmaniasis (subtype) - See Leishmaniasis
- Visceral myopathy - familial external ophthalmoplegia - See Familial visceral myopathy with external ophthalmoplegia
- Visceral myopathy familial - See Megaduodenum and/or megacystis
- Visceral myopathy, familial, with external ophthalmoplegia - See Familial visceral myopathy with external ophthalmoplegia
- Visceral neuromyopathy familial with pseudoobstruction megaduodenum barrett esophagus and cardiac abnormalities- See Mungan syndrome
- Visceral neuropathy familial
- Visceral steatosis
- Visual amnesia - See Agnosia
- Visual pathway and hypothalamic glioma, childhood
- Visual snow - See Visual snow syndrome
- Visual snow syndrome
- Vitamin A Deficiency - See Keratomalacia
- Vitamin A embryopathy
- Vitamin B1 deficiency - See Beriberi
- Vitamin B12-responsive methylmalonic acidemia
- Vitamin B12-responsive methylmalonic acidemia type cblA - See Methylmalonic aciduria, cblA type
- Vitamin B12-responsive methylmalonic acidemia type cblB - See Methylmalonic aciduria, cblB type
- Vitamin B12-responsive methylmalonic aciduria - See Vitamin B12-responsive methylmalonic acidemia
- Vitamin B12-responsive methylmalonic aciduria type cblA - See Methylmalonic aciduria, cblA type
- Vitamin B12-responsive methylmalonic aciduria, type cblB - See Methylmalonic aciduria, cblB type
- Vitamin B6 deficiency - See Pyridoxine deficiency
- Vitamin B6-dependent seizures - See Pyridoxine-dependent epilepsy
- Vitamin C deficiency - See Scurvy
- Vitamin D deficiency disease - See Rickets
- Vitamin deficiency, B6 - See Pyridoxine deficiency
- Vitamin D-Resistant Rickets, X-linked - See X-linked hypophosphatemia
- Vitamin K antagonists embryofetopathy - See Warfarin syndrome
- Vitamin K-antagonist embryofetopathy - See Warfarin syndrome
- Vitamin K-antagonist embryopathy - See Warfarin syndrome
- Vitamin-D deficiency rickets - See Rickets
- Vitelliform macular dystrophy type 2 - See Best vitelliform macular dystrophy
- Vitelliform macular dystrophy, adult-onset - See Adult-onset vitelliform macular dystrophy
- Vitelliform macular dystrophy, atypical - See Macular dystrophy, atypical vitelliform
- Vitiliginous choroiditis - See Birdshot chorioretinopathy
- Vitiligo - not a rare disease
- Vitiligo vulgaris, cleft palate, somatic and psychomotor retardation and facial dysmorphism - See Schrander-Stumpel Theunissen Hulsmans syndrome
- Vitiligo, psychomotor retardation, cleft palate and facial dysmorphism - See Schrander-Stumpel Theunissen Hulsmans syndrome
- Vitreoretinal degeneration
- Vitreoretinal degeneration, Snowflake type - See Snowflake vitreoretinal degeneration
- Vitreoretinochoroidopathy dominant - See Autosomal dominant vitreoretinochoroidopathy
- Vitritis - See Pars planitis
- Vitsala - See Kifafa seizure disorder
- VKC - See Vernal keratoconjunctivitis
- VKH disease - See Vogt-Koyanagi-Harada disease
- VKH syndrome - See Vogt-Koyanagi-Harada disease
- VLCAD deficiency
- VLCADD - See VLCAD deficiency
- VLDLRCH - See Dysequilibrium syndrome
- VMD1 - See Macular dystrophy, atypical vitelliform
- VMD2 - See Best vitelliform macular dystrophy
- Vocal cord dysfunction familial
- VODI - See Hepatic venoocclusive disease with immunodeficiency
- Vogt Spielmeyer disease - See Neuronal ceroid lipofuscinosis 3
- Vogt-Koyanagi-Harada disease
- Vogt-Koyanagi-Harada syndrome - See Vogt-Koyanagi-Harada disease
- Vohwinkel syndrome
- Voiding dysfunction and polycystic ovaries - See Fowler's syndrome
- Volcke Soekarman syndrome - See Macrocephaly-short stature-paraplegia syndrome
- Volcke-Soekarman syndrome - See Macrocephaly-short stature-paraplegia syndrome
- Volubilis syndrome - See Morning glory syndrome
- Von Bogaert-Bertrand disease - See Canavan disease
- Von Economo’s disease - See Encephalitis lethargica
- Von Eulenburg paramyotonia congenita - See Paramyotonia congenita
- Von Gierke disease - See Glycogen storage disease type 1A
- Von Hippel-Lindau disease - See Von Hippel-Lindau disease
- Von Hippel-Lindau disease
- Von Hippel-Lindau syndrome - See Von Hippel-Lindau disease
- Von Recklinghausen disease - See Neurofibromatosis type 1
- Von Voss Cherstvoy syndrome - See DK phocomelia syndrome
- Von Willebrand disease - not a rare disease
- Von Willebrand disease, platelet type - See Pseudo-Von Willebrand disease
- Von Willebrand factor receptor deficiency - See Giant platelet syndrome
- Von Willebrand factor, deficiency - See Von Willebrand disease - not a rare disease
- Voorhoeve disease - See Osteopathia striata cranial sclerosis
- VP - See Variegate porphyria
- VRCP autosomal dominant - See Autosomal dominant vitreoretinochoroidopathy
- Vrolik disease - See Osteogenesis imperfecta
- Vrolik type of osteogenesis imperfecta - See Osteogenesis imperfecta type II
- VSR syndrome - See Herrmann Opitz arthrogryposis syndrome
- Vulvar cancer
- Vulvar dysesthesia localized in the vestibule - See Vulvar Vestibulitis Syndrome
- Vulvar Vestibulitis Syndrome
- VVS - See Vulvar Vestibulitis Syndrome
- VWD - See Von Willebrand disease - not a rare disease
- VWS - See Van der Woude syndrome
- VWS2 - See Van der Woude syndrome 2
List of FDA Orphan Drugs
GARD has information from the Food and Drug Administration (FDA) on treatments approved for rare diseases, known as orphan products/drugs. The Orphan Drug Act was passed in 1983 to give drug companies incentives to develop treatments for rare diseases. The FDA Office of Orphan Products Development determines if a drug qualifies as an orphan product. The following information comes from the FDA database of orphan drug designations and approvals. You can output the data into an Excel spreadsheet from the FDA website. More information on orphan drug development is available from Global Genes and Orphanet.
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