200 rare diseases known to modern medicine
- Castleman Disease: A rare disorder in which benign (not cancer) growths form in lymph node tissue.
- Fibrodysplasia Ossificans Progressiva (FOP): A rare genetic disorder characterized by the abnormal growth of bone in soft tissues.
- Hutchinson-Gilford Progeria Syndrome: A rare genetic condition causing rapid aging in children.
- Stiff Person Syndrome: A rare neurological disorder characterized by severe muscle stiffness and spasms.
- Aquagenic Urticaria: A rare condition where individuals develop hives upon contact with water.
- Paraneoplastic Pemphigus: A rare autoimmune blistering disorder associated with underlying cancer.
- Harlequin Ichthyosis: A severe genetic skin disorder resulting in thickened, diamond-shaped scales.
- Maple Syrup Urine Disease: A rare metabolic disorder affecting the breakdown of certain amino acids.
- Proteus Syndrome: A rare genetic disorder characterized by overgrowth of bones, skin, and other tissues.
- Trimethylaminuria (Fish Odor Syndrome): A rare metabolic disorder causing a strong body odor resembling fish.
- Alkaptonuria (Black Urine Disease): A rare genetic disorder causing the accumulation of homogentisic acid in the body.
- Microcephaly: A rare neurological condition characterized by an abnormally small head and underdeveloped brain.
- Werewolf Syndrome (Hypertrichosis): A rare condition characterized by excessive hair growth over the body.
- Argyria (Blue Skin Syndrome): A rare condition causing skin to turn blue or gray due to exposure to silver compounds.
- Foreign Accent Syndrome: A rare speech disorder causing individuals to speak with an accent that is not their own.
- Moebius Syndrome: A rare neurological disorder affecting facial muscles, causing facial paralysis and limited eye movement.
- Epidermodysplasia Verruciformis (Tree Man Syndrome): A rare genetic disorder causing wart-like lesions and abnormal skin growths.
- Trichotillomania (Hair-Pulling Disorder): A rare mental health disorder characterized by compulsive hair pulling.
- Alice in Wonderland Syndrome: A rare neurological condition causing distorted perceptions of body size and shape.
- Fibrous Dysplasia: A rare bone disorder where fibrous tissue replaces normal bone, weakening the affected area.
- Capgras Syndrome: A rare psychiatric disorder where individuals believe that a loved one has been replaced by an imposter.
- Aicardi Syndrome: A rare genetic disorder characterized by the absence of the corpus callosum, seizures, and developmental delays.
- Berardinelli-Seip Congenital Lipodystrophy: A rare metabolic disorder characterized by the absence of body fat and metabolic complications.
- Cystinosis: A rare metabolic disorder where the amino acid cystine accumulates in cells, leading to organ damage.
- Evans Syndrome: A rare autoimmune disorder characterized by the destruction of red blood cells, platelets, and sometimes white blood cells.
- Hemophagocytic Lymphohistiocytosis (HLH): A rare immune system disorder characterized by the overactivation of immune cells, leading to organ damage.
- Idiopathic Intracranial Hypertension (IIH): A rare condition characterized by increased pressure within the skull, leading to symptoms like headaches and vision problems.
- Joubert Syndrome: A rare genetic disorder characterized by abnormal brain development, resulting in intellectual disability, movement disorders, and other symptoms.
- Kikuchi-Fujimoto Disease: A rare autoimmune disorder characterized by lymph node inflammation, fever, and fatigue.
- Lipoid Proteinosis: A rare genetic disorder characterized by skin and mucous membrane abnormalities, hoarse voice, and neurological symptoms.
- Maffucci Syndrome: A rare genetic disorder characterized by benign tumors of cartilage (enchondromas) and soft tissue, along with areas of abnormal skin pigmentation.
- Nakajo-Nishimura Syndrome: A rare autoinflammatory disorder characterized by periodic fever, lipodystrophy, joint contractures, and a distinctive skin rash.
- Ondine’s Curse (Congenital Central Hypoventilation Syndrome): A rare disorder characterized by abnormal control of breathing during sleep, leading to respiratory failure.
- Pantothenate Kinase-Associated Neurodegeneration (PKAN): A rare genetic disorder characterized by progressive neurodegeneration, movement disorders, and cognitive decline.
- Q Fever: A rare bacterial infection caused by Coxiella burnetii, leading to flu-like symptoms, pneumonia, and chronic fatigue.
- Russell-Silver Syndrome: A rare genetic disorder characterized by growth delays, distinctive facial features, and other physical abnormalities.
- Susac Syndrome: A rare autoimmune disorder characterized by inflammation and blockage of small blood vessels in the brain, retina, and inner ear, leading to neurological and sensory symptoms.
- Trisomy 18 (Edwards Syndrome): A rare chromosomal disorder characterized by multiple congenital abnormalities, developmental delays, and a high mortality rate.
- Urticarial Vasculitis: A rare form of vasculitis characterized by recurrent episodes of hives (urticaria) and inflammation of small blood vessels (vasculitis).
- Vanishing White Matter Disease: A rare genetic disorder characterized by progressive destruction of the brain’s white matter, leading to neurological deterioration and loss of motor function.
- Whipple’s Disease: A rare infectious disease caused by Tropheryma whipplei bacteria, leading to gastrointestinal symptoms, joint pain, and neurological problems.
- Xeroderma Pigmentosum: A rare genetic disorder characterized by extreme sensitivity to ultraviolet (UV) light, leading to sunburn, skin cancer, and eye problems.
- Yellow Nail Syndrome: A rare condition characterized by yellow, thickened nails, respiratory symptoms, and lymphedema.
- Zollinger-Ellison Syndrome: A rare disorder characterized by gastrin-secreting tumors in the pancreas or duodenum, leading to excessive gastric acid production and peptic ulcers.
- Aarskog-Scott Syndrome: A rare genetic disorder characterized by distinctive facial features, short stature, and skeletal abnormalities.
- Behçet’s Disease: A rare autoimmune disorder characterized by recurrent mouth ulcers, genital ulcers, and inflammation of the eyes and skin.
- Congenital Insensitivity to Pain with Anhidrosis (CIPA): A rare genetic disorder characterized by the inability to feel pain, temperature, or touch, along with the inability to sweat.
- Donohue Syndrome (Leprechaunism): A rare genetic disorder characterized by severe insulin resistance, leading to abnormal growth, facial features, and developmental delays.
- Ectodermal Dysplasia: A rare genetic disorder characterized by abnormalities in structures derived from the embryonic ectoderm, such as hair, teeth, nails, and sweat glands.
- Fanconi Anemia: A rare genetic disorder characterized by bone marrow failure, developmental abnormalities, and an increased risk of cancer.
- Griscelli Syndrome: A rare genetic disorder characterized by pigmentary dilution of the skin and hair, along with immunodeficiency and neurological abnormalities.
- Holt-Oram Syndrome: A rare genetic disorder characterized by heart defects and abnormalities of the upper limbs.
- Inclusion Body Myositis: A rare inflammatory muscle disease characterized by progressive muscle weakness and atrophy.
- Jansen’s Metaphyseal Chondrodysplasia: A rare genetic disorder characterized by abnormal bone development, short stature, and skeletal abnormalities.
- Kearns-Sayre Syndrome: A rare mitochondrial disorder characterized by progressive weakness of the eye muscles, heart conduction defects, and other neurological symptoms.
- Lipodystrophy Syndromes: A group of rare disorders characterized by abnormal distribution of body fat, metabolic abnormalities, and other complications.
- Menkes Disease: A rare genetic disorder characterized by impaired copper absorption, leading to developmental delays, neurological abnormalities, and distinctive physical features.
- Nephrogenic Systemic Fibrosis (NSF): A rare condition characterized by fibrosis of the skin and internal organs, usually associated with exposure to gadolinium-based contrast agents used in MRI scans.
- Osteogenesis Imperfecta: A rare genetic disorder characterized by brittle bones that are prone to fractures, along with other skeletal abnormalities.
- Pseudoxanthoma Elasticum: A rare genetic disorder characterized by abnormal mineralization of elastic fibers in the skin, eyes, and blood vessels.
- Rett Syndrome: A rare genetic disorder characterized by developmental regression, loss of motor skills, seizures, and distinctive hand movements.
- Shprintzen-Goldberg Syndrome: A rare genetic disorder characterized by craniofacial abnormalities, skeletal abnormalities, and other physical and developmental features.
- Tuberous Sclerosis Complex (TSC): A rare genetic disorder characterized by the growth of noncancerous tumors in multiple organs, including the brain, skin, kidneys, and heart.
- Uncombable Hair Syndrome: A rare genetic disorder characterized by dry, frizzy hair that is difficult to comb or style.
- Von Hippel-Lindau Disease: A rare genetic disorder characterized by the formation of tumors and cysts in multiple organs, particularly the brain, spinal cord, kidneys, and adrenal glands.
- Williams Syndrome: A rare genetic disorder characterized by cardiovascular abnormalities, distinctive facial features, developmental delays, and a friendly personality.
- X-linked Agammaglobulinemia: A rare genetic disorder characterized by a deficiency of B cells and immunoglobulins, leading to recurrent bacterial infections.
- Young Syndrome: A rare condition characterized by chronic sinusitis, bronchiectasis, and infertility in males.
- Abetalipoproteinemia: A rare genetic disorder characterized by the inability to absorb dietary fats and fat-soluble vitamins, leading to neurological and visual problems.
- Basal Cell Nevus Syndrome (Gorlin Syndrome): A rare genetic disorder characterized by the development of multiple basal cell carcinomas and other tumors.
- Cockayne Syndrome: A rare genetic disorder characterized by growth failure, neurological abnormalities, premature aging, and photosensitivity.
- Dubowitz Syndrome: A rare genetic disorder characterized by growth delays, facial abnormalities, and intellectual disability.
- Ectopia Cordis: A rare congenital condition characterized by the heart being located outside the chest cavity.
- Floating Harbor Syndrome: A rare genetic disorder characterized by short stature, delayed bone age, speech delays, and distinctive facial features.
- Greig Cephalopolysyndactyly Syndrome: A rare genetic disorder characterized by abnormal skull and facial features, extra fingers or toes, and intellectual disability.
- Hypophosphatasia: A rare genetic disorder characterized by defective mineralization of bones and teeth, leading to skeletal abnormalities and dental problems.
- Juvenile Polyposis Syndrome: A rare genetic disorder characterized by the development of multiple polyps in the gastrointestinal tract, leading to an increased risk of cancer.
- Klippel-Trenaunay Syndrome: A rare congenital condition characterized by abnormal development of blood vessels, soft tissue overgrowth, and bone and joint abnormalities.
- Lennox-Gastaut Syndrome: A rare form of epilepsy characterized by multiple seizure types, developmental delays, and cognitive impairment.
- Marshall Syndrome: A rare genetic disorder characterized by distinctive facial features, hearing loss, and skeletal abnormalities.
- Nephronophthisis: A rare genetic disorder characterized by the progressive destruction of kidney tubules, leading to kidney failure.
- Opitz G/BBB Syndrome: A rare genetic disorder characterized by midline defects, including cleft lip and palate, heart defects, and intellectual disability.
- Phelan-McDermid Syndrome: A rare genetic disorder characterized by developmental delays, intellectual disability, speech deficits, and distinctive facial features.
- Rhabdoid Tumor Predisposition Syndrome: A rare genetic disorder characterized by an increased risk of developing aggressive tumors, particularly rhabdoid tumors.
- Schinzel-Giedion Syndrome: A rare genetic disorder characterized by distinctive facial features, developmental delays, seizures, and skeletal abnormalities.
- Trisomy 13 (Patau Syndrome): A rare chromosomal disorder characterized by multiple congenital abnormalities, intellectual disability, and a high mortality rate.
- Ulnar-Mammary Syndrome: A rare genetic disorder characterized by abnormalities of the ulnar bone, mammary glands, teeth, and reproductive system.
- VACTERL Association: A rare condition characterized by multiple congenital abnormalities, including vertebral defects, anal atresia, cardiac defects, tracheoesophageal fistula, renal anomalies, and limb abnormalities.
- Wolfram Syndrome: A rare genetic disorder characterized by diabetes mellitus, optic atrophy, deafness, and neurological abnormalities.
- X-linked Hypohidrotic Ectodermal Dysplasia: A rare genetic disorder characterized by abnormalities of the skin, hair, teeth, and sweat glands due to a deficiency of sweat glands.
- Alagille Syndrome: A rare genetic disorder characterized by liver abnormalities, heart defects, distinctive facial features, and other physical and developmental abnormalities.
- Amyloidosis: A rare disorder characterized by the accumulation of abnormal protein deposits (amyloid) in tissues and organs, leading to organ dysfunction and failure.
- Bartter Syndrome: A rare genetic disorder characterized by electrolyte imbalances, renal salt wasting, and metabolic alkalosis.
- Chronic Recurrent Multifocal Osteomyelitis (CRMO): A rare inflammatory bone disorder characterized by recurrent episodes of bone pain and inflammation in multiple sites.
- Dysautonomia: A rare disorder affecting the autonomic nervous system, leading to dysfunction in regulating bodily functions such as heart rate, blood pressure, digestion, and temperature.
- Eosinophilic Granulomatosis with Polyangiitis (Churg-Strauss Syndrome): A rare autoimmune disorder characterized by inflammation of small and medium-sized blood vessels, leading to organ damage.
- Fibrosing Mediastinitis: A rare condition characterized by the abnormal growth of fibrous tissue in the mediastinum, leading to compression of nearby structures.
- Glutaric Acidemia Type 1: A rare metabolic disorder characterized by the deficiency of an enzyme involved in the breakdown of certain amino acids, leading to neurological damage.
- Hemimegalencephaly: A rare neurological disorder characterized by abnormal brain development, leading to seizures, developmental delays, and other neurological symptoms.
- I-cell Disease (Mucolipidosis II): A rare lysosomal storage disorder characterized by the accumulation of glycoproteins in cells, leading to skeletal abnormalities, facial features, and developmental delays.
- Jansen’s Metaphyseal Chondrodysplasia: A rare genetic disorder characterized by abnormal bone development, short stature, and skeletal abnormalities.
- Kearns-Sayre Syndrome: A rare mitochondrial disorder characterized by progressive weakness of the eye muscles, heart conduction defects, and other neurological symptoms.
- Langerhans Cell Histiocytosis: A rare disorder characterized by the abnormal proliferation of Langerhans cells, leading to the formation of tumors or lesions in various organs.
- Marshall Syndrome: A rare genetic disorder characterized by distinctive facial features, hearing loss, and skeletal abnormalities.
- Nevoid Basal Cell Carcinoma Syndrome (Gorlin Syndrome): A rare genetic disorder characterized by the development of multiple basal cell carcinomas and other tumors.
- Opsismodysplasia: A rare genetic disorder characterized by short stature, facial abnormalities, and skeletal abnormalities.
- Pfeiffer Syndrome: A rare genetic disorder characterized by craniosynostosis, distinctive facial features, and abnormalities of the hands and feet.
- Q Fever: A rare bacterial infection caused by Coxiella burnetii, leading to flu-like symptoms, pneumonia, and chronic fatigue.
- Rothmund-Thomson Syndrome: A rare genetic disorder characterized by skin abnormalities, skeletal abnormalities, and an increased risk of cancer.
- Smith-Lemli-Opitz Syndrome: A rare genetic disorder characterized by developmental delays, distinctive facial features, and multiple congenital abnormalities.
- Takayasu’s Arteritis: A rare autoimmune disorder characterized by inflammation of the large blood vessels, leading to narrowing or blockage of the arteries.
- Urticarial Vasculitis: A rare form of vasculitis characterized by recurrent episodes of hives (urticaria) and inflammation of small blood vessels (vasculitis).
- Vanishing White Matter Disease: A rare genetic disorder characterized by progressive destruction of the brain’s white matter, leading to neurological deterioration and loss of motor function.
- Williams Syndrome: A rare genetic disorder characterized by cardiovascular abnormalities, distinctive facial features, developmental delays, and a friendly personality.
- X-linked Agammaglobulinemia: A rare genetic disorder characterized by a deficiency of B cells and immunoglobulins, leading to recurrent bacterial infections.
- Young Syndrome: A rare condition characterized by chronic sinusitis, bronchiectasis, and infertility in males.
- Zollinger-Ellison Syndrome: A rare disorder characterized by gastrin-secreting tumors in the pancreas or duodenum, leading to excessive gastric acid production and peptic ulcers.
- Aarskog-Scott Syndrome: A rare genetic disorder characterized by distinctive facial features, short stature, and skeletal abnormalities.
- Behçet’s Disease: A rare autoimmune disorder characterized by recurrent mouth ulcers, genital ulcers, and inflammation of the eyes and skin.
- Congenital Insensitivity to Pain with Anhidrosis (CIPA): A rare genetic disorder characterized by the inability to feel pain, temperature, or touch, along with the inability to sweat.
- Donohue Syndrome (Leprechaunism): A rare genetic disorder characterized by severe insulin resistance, leading to abnormal growth, facial features, and developmental delays.
- Ectodermal Dysplasia: A rare genetic disorder characterized by abnormalities in structures derived from the embryonic ectoderm, such as hair, teeth, nails, and sweat glands.
- Fanconi Anemia: A rare genetic disorder characterized by bone marrow failure, developmental abnormalities, and an increased risk of cancer.
- Griscelli Syndrome: A rare genetic disorder characterized by pigmentary dilution of the skin and hair, along with immunodeficiency and neurological abnormalities.
- Holt-Oram Syndrome: A rare genetic disorder characterized by heart defects and abnormalities of the upper limbs.
- Inclusion Body Myositis: A rare inflammatory muscle disease characterized by progressive muscle weakness and atrophy.
- Jansen’s Metaphyseal Chondrodysplasia: A rare genetic disorder characterized by abnormal bone development, short stature, and skeletal abnormalities.
- Kearns-Sayre Syndrome: A rare mitochondrial disorder characterized by progressive weakness of the eye muscles, heart conduction defects, and other neurological symptoms.
- Lipoid Proteinosis: A rare genetic disorder characterized by skin and mucous membrane abnormalities, hoarse voice, and neurological symptoms.
- Marshall Syndrome: A rare genetic disorder characterized by distinctive facial features, hearing loss, and skeletal abnormalities.
- Nakajo-Nishimura Syndrome: A rare autoinflammatory disorder characterized by periodic fever, lipodystrophy, joint contractures, and a distinctive skin rash.
- Opitz G/BBB Syndrome: A rare genetic disorder characterized by midline defects, including cleft lip and palate, heart defects, and intellectual disability.
- Phelan-McDermid Syndrome: A rare genetic disorder characterized by developmental delays, intellectual disability, speech deficits, and distinctive facial features.
- Q Fever: A rare bacterial infection caused by Coxiella burnetii, leading to flu-like symptoms, pneumonia, and chronic fatigue.
- Rothmund-Thomson Syndrome: A rare genetic disorder characterized by skin abnormalities, skeletal abnormalities, and an increased risk of cancer.
- Susac Syndrome: A rare autoimmune disorder characterized by inflammation and blockage of small blood vessels in the brain, retina, and inner ear, leading to neurological and sensory symptoms.
- Trisomy 13 (Patau Syndrome): A rare chromosomal disorder characterized by multiple congenital abnormalities, intellectual disability, and a high mortality rate.
- Ulnar-Mammary Syndrome: A rare genetic disorder characterized by abnormalities of the ulnar bone, mammary glands, teeth, and reproductive system.
- VACTERL Association: A rare condition characterized by multiple congenital abnormalities, including vertebral defects, anal atresia, cardiac defects, tracheoesophageal fistula, renal anomalies, and limb abnormalities.
- Wolfram Syndrome: A rare genetic disorder characterized by diabetes mellitus, optic atrophy, deafness, and neurological abnormalities.
- X-linked Hypohidrotic Ectodermal Dysplasia: A rare genetic disorder characterized by abnormalities of the skin, hair, teeth, and sweat glands due to a deficiency of sweat glands.
- Young Syndrome: A rare condition characterized by chronic sinusitis, bronchiectasis, and infertility in males.
- Zollinger-Ellison Syndrome: A rare disorder characterized by gastrin-secreting tumors in the pancreas or duodenum, leading to excessive gastric acid production and peptic ulcers.
- Aarskog-Scott Syndrome: A rare genetic disorder characterized by distinctive facial features, short stature, and skeletal abnormalities.
- Behçet’s Disease: A rare autoimmune disorder characterized by recurrent mouth ulcers, genital ulcers, and inflammation of the eyes and skin.
- Congenital Insensitivity to Pain with Anhidrosis (CIPA): A rare genetic disorder characterized by the inability to feel pain, temperature, or touch, along with the inability to sweat.
- Donohue Syndrome (Leprechaunism): A rare genetic disorder characterized by severe insulin resistance, leading to abnormal growth, facial features, and developmental delays.
- Ectodermal Dysplasia: A rare genetic disorder characterized by abnormalities in structures derived from the embryonic ectoderm, such as hair, teeth, nails, and sweat glands.
- Fanconi Anemia: A rare genetic disorder characterized by bone marrow failure, developmental abnormalities, and an increased risk of cancer.
- Griscelli Syndrome: A rare genetic disorder characterized by pigmentary dilution of the skin and hair, along with immunodeficiency and neurological abnormalities.
- Holt-Oram Syndrome: A rare genetic disorder characterized by heart defects and abnormalities of the upper limbs.
- Inclusion Body Myositis: A rare inflammatory muscle disease characterized by progressive muscle weakness and atrophy.
- Jansen’s Metaphyseal Chondrodysplasia: A rare genetic disorder characterized by abnormal bone development, short stature, and skeletal abnormalities.
- Kearns-Sayre Syndrome: A rare mitochondrial disorder characterized by progressive weakness of the eye muscles, heart conduction defects, and other neurological symptoms.
- Lipoid Proteinosis: A rare genetic disorder characterized by skin and mucous membrane abnormalities, hoarse voice, and neurological symptoms.
- Marshall Syndrome: A rare genetic disorder characterized by distinctive facial features, hearing loss, and skeletal abnormalities.
- Nakajo-Nishimura Syndrome: A rare autoinflammatory disorder characterized by periodic fever, lipodystrophy, joint contractures, and a distinctive skin rash.
- Opitz G/BBB Syndrome: A rare genetic disorder characterized by midline defects, including cleft lip and palate, heart defects, and intellectual disability.
- Phelan-McDermid Syndrome: A rare genetic disorder characterized by developmental delays, intellectual disability, speech deficits, and distinctive facial features.
- Q Fever: A rare bacterial infection caused by Coxiella burnetii, leading to flu-like symptoms, pneumonia, and chronic fatigue.
- Rothmund-Thomson Syndrome: A rare genetic disorder characterized by skin abnormalities, skeletal abnormalities, and an increased risk of cancer.
- Susac Syndrome: A rare autoimmune disorder characterized by inflammation and blockage of small blood vessels in the brain, retina, and inner ear, leading to neurological and sensory symptoms.
- Trisomy 13 (Patau Syndrome): A rare chromosomal disorder characterized by multiple congenital abnormalities, intellectual disability, and a high mortality rate.
- Ulnar-Mammary Syndrome: A rare genetic disorder characterized by abnormalities of the ulnar bone, mammary glands, teeth, and reproductive system.
- VACTERL Association: A rare condition characterized by multiple congenital abnormalities, including vertebral defects, anal atresia, cardiac defects, tracheoesophageal fistula, renal anomalies, and limb abnormalities.
- Wolfram Syndrome: A rare genetic disorder characterized by diabetes mellitus, optic atrophy, deafness, and neurological abnormalities.
- X-linked Hypohidrotic Ectodermal Dysplasia: A rare genetic disorder characterized by abnormalities of the skin, hair, teeth, and sweat glands due to a deficiency of sweat glands.
- Young Syndrome: A rare condition characterized by chronic sinusitis, bronchiectasis, and infertility in males.
- Zollinger-Ellison Syndrome: A rare disorder characterized by gastrin-secreting tumors in the pancreas or duodenum, leading to excessive gastric acid production and peptic ulcers.
- Aarskog-Scott Syndrome: A rare genetic disorder characterized by distinctive facial features, short stature, and skeletal abnormalities.
- Behçet’s Disease: A rare autoimmune disorder characterized by recurrent mouth ulcers, genital ulcers, and inflammation of the eyes and skin.
- Congenital Insensitivity to Pain with Anhidrosis (CIPA): A rare genetic disorder characterized by the inability to feel pain, temperature, or touch, along with the inability to sweat.
- Donohue Syndrome (Leprechaunism): A rare genetic disorder characterized by severe insulin resistance, leading to abnormal growth, facial features, and developmental delays.
- Ectodermal Dysplasia: A rare genetic disorder characterized by abnormalities in structures derived from the embryonic ectoderm, such as hair, teeth, nails, and sweat glands.
- Fanconi Anemia: A rare genetic disorder characterized by bone marrow failure, developmental abnormalities, and an increased risk of cancer.
- Griscelli Syndrome: A rare genetic disorder characterized by pigmentary dilution of the skin and hair, along with immunodeficiency and neurological abnormalities.
- Holt-Oram Syndrome: A rare genetic disorder characterized by heart defects and abnormalities of the upper limbs.
- Inclusion Body Myositis: A rare inflammatory muscle disease characterized by progressive muscle weakness and atrophy.
- Jansen’s Metaphyseal Chondrodysplasia: A rare genetic disorder characterized by abnormal bone development, short stature, and skeletal abnormalities.
- Kearns-Sayre Syndrome: A rare mitochondrial disorder characterized by progressive weakness of the eye muscles, heart conduction defects, and other neurological symptoms.
- Lipoid Proteinosis: A rare genetic disorder characterized by skin and mucous membrane abnormalities, hoarse voice, and neurological symptoms.
- Marshall Syndrome: A rare genetic disorder characterized by distinctive facial features, hearing loss, and skeletal abnormalities.
- Nakajo-Nishimura Syndrome: A rare autoinflammatory disorder characterized by periodic fever, lipodystrophy, joint contractures, and a distinctive skin rash.
- Opitz G/BBB Syndrome: A rare genetic disorder characterized by midline defects, including cleft lip and palate, heart defects, and intellectual disability.
- Phelan-McDermid Syndrome: A rare genetic disorder characterized by developmental delays, intellectual disability, speech deficits, and distinctive facial features.
- Q Fever: A rare bacterial infection caused by Coxiella burnetii, leading to flu-like symptoms, pneumonia, and chronic fatigue.
- Rothmund-Thomson Syndrome: A rare genetic disorder characterized by skin abnormalities, skeletal abnormalities, and an increased risk of cancer.
- Susac Syndrome: A rare autoimmune disorder characterized by inflammation and blockage of small blood vessels in the brain, retina, and inner ear, leading to neurological and sensory symptoms.
- Trisomy 13 (Patau Syndrome): A rare chromosomal disorder characterized by multiple congenital abnormalities, intellectual disability, and a high mortality rate.
- Ulnar-Mammary Syndrome: A rare genetic disorder characterized by abnormalities of the ulnar bone, mammary glands, teeth, and reproductive system.
- VACTERL Association: A rare condition characterized by multiple congenital abnormalities, including vertebral defects, anal atresia, cardiac defects, tracheoesophageal fistula, renal anomalies, and limb abnormalities.
- Wolfram Syndrome: A rare genetic disorder characterized by diabetes mellitus, optic atrophy, deafness, and neurological abnormalities.
- X-linked Hypohidrotic Ectodermal Dysplasia: A rare genetic disorder characterized by abnormalities of the skin, hair, teeth, and sweat glands due to a deficiency of sweat glands.
- Young Syndrome: A rare condition characterized by chronic sinusitis, bronchiectasis, and infertility in males.
- Zollinger-Ellison Syndrome: A rare disorder characterized by gastrin-secreting tumors in the pancreas or duodenum, leading to excessive gastric acid production and peptic ulcers.
- Aarskog-Scott Syndrome: A rare genetic disorder characterized by distinctive facial features, short stature, and skeletal abnormalities.
- Behçet’s Disease: A rare autoimmune disorder characterized by recurrent mouth ulcers, genital ulcers, and inflammation of the eyes and skin.
- Congenital Insensitivity to Pain with Anhidrosis (CIPA): A rare genetic disorder characterized by the inability to feel pain, temperature, or touch, along with the inability to sweat.
- Donohue Syndrome (Leprechaunism): A rare genetic disorder characterized by severe insulin resistance, leading to abnormal growth, facial features, and developmental delays.
- Ectodermal Dysplasia: A rare genetic disorder characterized by abnormalities in structures derived from the embryonic ectoderm, such as hair, teeth, nails, and sweat glands.