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US non-profit biotechnology company
Cure Rare Disease is a non-profit biotechnology company based in Woodbridge, Connecticut that is working to create novel therapeutics using gene therapy
Cure_Rare_Disease
Disease affecting a small percentage of the population
rare disease is any disease that affects a small percentage of the population. In some parts of the world, the term orphan disease describes a rare disease
Rare_disease
American biotechnology company
Retrieved 2023-08-25. "Cure Rare Disease Collaboration with Taconic Biosciences Produces Novel ADSSL1 Mouse Model | | Cure Rare Disease". www.cureraredisease
Taconic_Biosciences
Orphan Diseases (WAO(R)D) The World Association of Cured Rare Diseases (WACRD) Sciensano: Rare diseases Rare Disease Day Belgium Rare Diseases Belgium
List of rare disease organisations
List_of_rare_disease_organisations
Terminal recessive genetic condition
Lafora disease is a rare, autosomal recessive genetic disorder which results in myoclonic epilepsy and usually results in death several years after the
Lafora_disease
Medical condition
(SBMA), popularly known as Kennedy's disease, is a rare, adult-onset, X-linked recessive lower motor neuron disease caused by trinucleotide CAG repeat expansions
Spinal and bulbar muscular atrophy
Spinal_and_bulbar_muscular_atrophy
Medical condition
Whipple's disease is a rare systemic infectious disease caused by the bacterium Tropheryma whipplei. First described by George Hoyt Whipple in 1907 and
Whipple's_disease
Rare neurodegenerative disease caused by prions
a rare, incurable, and fatal neurodegenerative disorder that was formerly common among the Fore people of Papua New Guinea. It was a prion disease that
Kuru_(disease)
Medical condition
became rare following occupational exposure limits established around 1950. Berylliosis is an occupational lung disease. While there is no cure, symptoms
Berylliosis
Neurodegenerative disorder
Canavan disease, or Canavan–Van Bogaert–Bertrand disease, is a rare and fatal autosomal recessive degenerative disease that causes progressive damage to
Canavan_disease
Rare neuromuscular disease
electronic speaking aids. There is no known cure or treatment. As of 2014, the disease is of unknown etiology. The disease has had no apparent effect on the twins'
Fields_condition
Degenerative neurological disorder
dominant manner. In rare instances, exposure to brain or spinal tissue from an infected person has resulted in transmission of disease, and a variant form
Creutzfeldt–Jakob_disease
American immunologist, author (born 1985)
Couzin-Frankel, Jennifer (2016-07-11). "A young doctor fights to cure his own rare, deadly disease". Science. Retrieved 20 July 2019. Thomas, Katie (February
David_Fajgenbaum
Medical condition
hyperkeratosis. Meleda Disease is a skin condition which usually can be identified not long after birth. This is a genetic condition but it is very rare. The hands
Meleda_disease
Disease affecting bone remodeling
treatments of Paget's disease can vary. Although there is no cure for Paget's disease, medications (bisphosphonates and calcitonin) can help control
Paget's_disease_of_bone
Progressive neurodegenerative disease
Parkinson's disease (PD), or simply Parkinson's, is a neurodegenerative disease primarily of the central nervous system, affecting both motor and non-motor
Parkinson's_disease
Lymphedema commonly located in the legs
system. Milroy's disease is also known as primary or hereditary lymphedema type 1A or early onset lymphedema. It is a very rare disease with only about
Milroy's_disease
Rare medical conditions that result in easy blistering of the skin and mucous membranes
painful. Its severity can range from mild to fatal. Inherited EB is a rare disease, with a prevalence of 8.2 per million live births in the United States
Epidermolysis_bullosa
Condition negatively affecting an organism
phenomenon. Incurable disease A disease that cannot be cured. Incurable diseases are not necessarily terminal diseases, and sometimes a disease's symptoms can
Disease
Progressive neurological disease
degenerative. There is no known effective treatment or cure. SCA can affect anyone of any age. The disease is caused by either a recessive or dominant gene
Spinocerebellar_ataxia
Rare skeletal genetic disorder
Camurati–Engelmann disease (CED) is a very rare autosomal dominant genetic disorder that causes characteristic anomalies in the skeleton. It is also known
Camurati–Engelmann_disease
Medical condition
Subacute sclerosing panencephalitis (SSPE), also known as Dawson disease, is a rare form of progressive brain inflammation caused by a persistent infection
Subacute sclerosing panencephalitis
Subacute_sclerosing_panencephalitis
Fatal childhood genetic condition
precise function of the CLN3 gene product remains unknown. Batten disease is rare; misdiagnosis may lead to increased medical expenses, family stress
Batten_disease
Group of lymphoproliferative disorders
Castleman disease (CD) describes a group of rare lymphoproliferative disorders that involve enlarged lymph nodes, and a broad range of inflammatory symptoms
Castleman_disease
Inflammatory disorder
floaters or visual field defects. Optic nerve involvement in Behçet's disease is rare, typically presenting as progressive optic atrophy and visual loss
Behçet's_disease
Rare neurodegenerative disease
(ALS), also known as motor neuron disease (MND) or Lou Gehrig's disease, is a rare terminal neurodegenerative disease defined by the progressive loss of
ALS
Medical condition
Jay Frank Schamberg, who described it in 1901. There is no known cure for this disease but it is not a life-threatening condition and is mainly of cosmetic
Schamberg_disease
Medical condition
prevent misdiagnosis and inappropriate treatment. Kikuchi's disease is a very rare disease mainly seen in Japan. Isolated cases are reported in North America
Kikuchi_disease
Rare metabolism disorder
also known as mucopolysaccharidosis type III (MPS III), is a rare lifelong genetic disease that mainly affects the brain and spinal cord. It is caused
Sanfilippo_syndrome
Medical condition
Multisystem proteinopathy (MSP) is a rare, inherited degenerative disease characterized by progressive dysfunction across multiple organ systems, primarily
Multisystem_proteinopathy
Rare human genetic lysosomal storage disorder
Fabry disease, also known as Anderson–Fabry disease, is a rare genetic disease that can affect many parts of the body, including the kidneys, heart, brain
Fabry_disease
Disease in which fibrous connective tissue turns into bone
also called Münchmeyer disease or formerly myositis ossificans progressiva, is an extremely rare connective tissue disease. Fibrous connective tissue
Fibrodysplasia ossificans progressiva
Fibrodysplasia_ossificans_progressiva
American football player (born 1991)
Rothstein, Michael (August 29, 2018). "Lions' Zach Zenner working to cure rare disease, save kids' lives". ESPN.com. Archived from the original on September
Zach_Zenner
Type of inflammatory bowel disease
symptoms include irritable bowel syndrome and Behçet's disease. There is no known cure for Crohn's disease. Treatment options are intended to help with symptoms
Crohn's_disease
Medical condition
Krabbe disease (KD) (also known as globoid cell leukodystrophy or galactosylceramide lipidosis) is a rare and often fatal lysosomal storage disease that
Krabbe_disease
Progressive neurodegenerative disease
remain primarily research tools. There is no disease-modifying treatments proven to cure Alzheimer's disease, and because of this, AD research has focused
Alzheimer's_disease
Treatment of Parkinson's disease
general wellness maintenance, exercise, and nutrition. At present, no cure for the disease is known, but medications or surgery can provide relief from the
Management of Parkinson's disease
Management_of_Parkinson's_disease
American prion researcher
disease treatment and cure. In 2023, she earned the Paper of the Year Award from the Oligonucleotide Therapeutics Society. As of 2026, prion diseases
Sonia_M._Vallabh
Group of brain diseases induced by prions
Transmissible spongiform encephalopathies (TSEs), or prion diseases, are a group of rare, progressive, incurable, and invariably fatal conditions that
Transmissible spongiform encephalopathy
Transmissible_spongiform_encephalopathy
Tuberculosis of the spine
treatment for Pott's disease. Surgical intervention may be appropriate for some patients. With early intervention, Pott's disease can be cured and completely
Pott's_disease
Medical condition
comparison to men. Cases of Cushing's disease are rare, and little epidemiological data is available on the disease. An 18-year study conducted on the population
Cushing's_disease
Medical condition
Erdheim–Chester disease (ECD) is an extremely rare disease classified as a non-Langerhans-cell histiocytic neoplasm. In 2016, the World Health Organization
Erdheim–Chester_disease
Medical condition
Schindler disease, also known as Kanzaki disease and alpha-N-acetylgalactosaminidase deficiency, is a rare disease found in humans. This lysosomal storage
Schindler_disease
Rare recessive genetic disorder
Urbach–Wiethe disease is a very rare recessive genetic disorder, with approximately 400 reported cases since its discovery. It was first officially reported
Urbach–Wiethe_disease
Disorder of the inner ear
vestibular schwannoma, or a tumor of the endolymphatic sac. No cure for Ménière's disease is known, but medications, diet, physical therapy, counseling
Ménière's_disease
Human neurodegenerative disease
Gerstmann–Sträussler–Scheinker syndrome (GSS) is an extremely rare, invariably fatal neurodegenerative disease that usually affects patients from 35 to 55 years in
Gerstmann–Sträussler–Scheinker syndrome
Gerstmann–Sträussler–Scheinker_syndrome
Involuntary movement of the hands or feet
are many drugs that can control it, no cure has yet been identified. Historically, choreas like Huntington disease and Sydenham's chorea were called Saint
Chorea
Inherited neurodegenerative disorder
of European descent. It is rare among the Finnish and the Japanese, while the incidence in Africa is unknown. The disease affects males and females equally
Huntington's_disease
Rare, severe disease of lysosomal storage
ages of five and fifteen years. A rare form of this disease, known as Adult-Onset or Late-Onset Tay–Sachs disease, usually has its first symptoms during
Tay–Sachs_disease
Rare genetic disorder of the white matter of the brain
Alexander disease is a very rare autosomal dominant leukodystrophy, which are neurological conditions caused by anomalies in the myelin which protects
Alexander_disease
2019 American film by Ciarán Foy
The film follows a boy with a rare autoimmune disease who is taken by his parents to a private medical facility to be cured. Netflix released the film on
Eli_(2019_film)
Prion disease of the human brain
sporadic form of disease. The targeting of this mutation has been suggested as a strategy for treatment, or possibly as a cure for the disease. In late 1983
Fatal_insomnia
Autoimmune endocrine disease
Graves' disease, also known as toxic diffuse goiter or Basedow's disease, is an autoimmune disease that affects the thyroid. It frequently results in,
Graves'_disease
Genetic multisystem copper-transport disease
cardiomyopathy (weakness of the heart muscle) is a rare but recognized problem in Wilson's disease; it may lead to heart failure (fluid accumulation due
Wilson's_disease
Medical condition
or similar support through most of their lives, but this is rare. Dejerine–Sottas disease is characterized by moderate to severe lower and upper extremity
Dejerine–Sottas_disease
Autoimmune disorder
Coeliac disease (Commonwealth English) or celiac disease (American English) is a chronic autoimmune disease, mainly affecting the small intestine. It
Coeliac_disease
Disease resulting from a lack of vitamin C
and "certain herbs". The Dutch sailors in the area were said to cure the same disease by drinking lime juice. In 1614, John Woodall, Surgeon General of
Scurvy
Autoimmune disease
diagnostic test requires a lip biopsy. Neither a cure nor a specific treatment for Sjögren's disease is known to restore gland secretion. Instead, treatment
Sjögren's_disease
US nonprofit organization
nonprofit organization that raises money and awareness to help find a cure for rare ("nonsense") mutations of cystic fibrosis (CF), a genetic disorder that
Emily's_Entourage
Human infectious diseases may be characterized by their case fatality rate (CFR), the proportion of people diagnosed with a disease who die from it (cf
List of human disease case fatality rates
List_of_human_disease_case_fatality_rates
Medical condition of the brain
US: CurePSP, a nonprofit organization for promoting awareness, care and research of PSP, CBD, MSA "and other prime of life neurodegenerative diseases" In
Progressive supranuclear palsy
Progressive_supranuclear_palsy
Food preservation and flavouring processes
supplant it. While meat-preservation processes like curing were mainly developed in order to prevent disease and to increase food security, the advent of modern
Curing_(food_preservation)
6th episode of the 1st season of Fringe
concludes she was suffering from a rare and incurable disease, "Bellini's lymphocemia," but was mysteriously cured. Further tests reveal Kramer was held
The_Cure_(Fringe)
Blood cancer that affects B-type white blood cells
non-recurrence. Early-stage aggressive disease is treated with chemotherapy and often radiation, with a 70–90% cure rate. Late-stage indolent lymphomas are
B-cell_lymphoma
Infectious disease caused by Borrelia bacteria, spread by ticks
original or appearance of new signs of the disease, occurs only in a few people. Remaining people are considered cured but continue to experience subjective
Lyme_disease
Type of arthritis of the spine
There is no cure for AS, but treatments and medications can reduce symptoms and pain. Medications for AS may be broadly considered either "disease-modifying"
Ankylosing_spondylitis
Rare disease of the bones
'bone becoming stone', also known as marble bone disease or Albers-Schönberg disease, is an extremely rare inherited disorder whereby the bones harden, becoming
Osteopetrosis
Rare autosomal-recessive human disease
sheath. There is currently no cure for Friedreich's ataxia, but treatment focuses on symptom management and slowing disease progression. In 2023, the US
Friedreich's_ataxia
Medical condition
rashes. There is no known cure. While the term pemphigus typically refers to "a rare group of blistering autoimmune diseases" affecting "the skin and mucous
Hailey–Hailey_disease
Medical condition
Waldmann disease, also known as Primary Intestinal Lymphangiectasia (PIL), is a rare disease characterized by enlargement of the lymph vessels supplying
Waldmann_disease
Fungal infection
of disease. It may appear similar to aspergillosis. Treatment is generally with amphotericin B and surgical debridement. Mucormycosis is usually rare. A
Mucormycosis
Medical condition
from congenital central hypoventilation syndrome (CCHS). ROHHAD is a rare disease, with only 100 reported cases worldwide thus far. The first sign of ROHHAD
ROHHAD
Congress that would cut funds required for the production of drugs to cure rare diseases, but learns that passage of the bill will have fatal consequences
List of Highway to Heaven episodes
List_of_Highway_to_Heaven_episodes
Dementia involving the frontal or temporal lobes
features of FTD subtypes. There is no cure for FTD, nor are any disease-modifying treatments approved that could slow disease progression. The aim of treatment
Frontotemporal_dementia
Rare congenital neuromuscular disorder
neuron disease Distal spinal muscular atrophy type 1 Distal spinal muscular atrophy type 2 "Spinal muscular atrophy". Genetic and Rare Diseases Information
Spinal_muscular_atrophy
Skin infection between the buttocks
Pilonidal disease is a type of skin infection that typically occurs as a cyst between the cheeks of the buttocks and often at the upper end. Symptoms
Pilonidal_disease
Mammal parasitic disease
usually cure the disease if given shortly after the person is infected, but become less effective the longer a person has had Chagas disease. When used
Chagas_disease
Medical condition
Reticular dysgenesis (RD) is a rare, inherited autosomal recessive disease that results in immunodeficiency. Individuals with RD have mutations in both
Reticular_dysgenesis
American founder of Scleroderma Research Foundation (1953–2002)
the disease in the medical community, she went on to found the Scleroderma Research Foundation in 1987, an organization dedicated to finding a cure. According
Sharon_Monsky
American singer-songwriter (born 1978)
learning that his youngest daughter, Rose, had a rare genetic disease, he founded and runs the To Cure a Rose Foundation and is in the process of founding
Casey_McPherson
U.S. health institute
treatments and cures for both common and rare diseases. NIAMS' mission is to support the research of arthritis, musculoskeletal and skin diseases. The institute
National Institute of Arthritis and Musculoskeletal and Skin Diseases
National_Institute_of_Arthritis_and_Musculoskeletal_and_Skin_Diseases
Medical condition
Idea to Help His Friend with a Rare Disease Leads to Scientific Breakthroughs". 11 June 2019. "CureGSD1b Team - CureGSD1b". 8 May 2022. "Glycogen Metabolism"
Glycogen_storage_disease
Pathological condition that affects the vagina
A vaginal disease is a pathological condition that affects part or all of the vagina. Sexually transmitted infections that affect the vagina include:
Vaginal_disease
Medical condition
with Microcephaly and Intellectual Disability, is a very rare autosomal recessive genetic disease. Only a very limited number of cases have been reported
Filippi_syndrome
U.S. nonprofit organization
include funding research on treatment and cures for rare diseases; lobbying for legislation to benefit the rare diseases community (in addition to the Orphan
National Organization for Rare Disorders
National_Organization_for_Rare_Disorders
Type of blood and immune-system cancer
become and whether or not it has favorable features. If the disease is detected early, a cure is often possible. In the United States, 89% of people diagnosed
Hodgkin_lymphoma
Stiffening of a tissue or anatomical feature
a rare, chronic disease which affects the skin, and in some cases also blood vessels and internal organs. Tuberous sclerosis, a rare genetic disease which
Sclerosis_(medicine)
Rare genetic disorder involving childhood obesity and multiple organ dysfunction
(November 2021). "A review of Alström syndrome: a rare monogenic ciliopathy". Intractable & Rare Diseases Research. 10 (4): 257–262. doi:10.5582/irdr.2021
Alström_syndrome
Athlete, speaker
for a CURE". Toastmaster. Retrieved 2015-05-21. "Tampa at Center of Research to Cure Friedreich's Ataxia". Rare Daily. Global Genes - Allies in Rare Disease
Kyle_Bryant
Genetic disorder group
the disease currently involves enzyme replacement therapy and gene therapy is a possibility for the future, a solution which may cure the disease completely
Autophagic_vacuolar_myopathy
X-linked recessive copper-transport disorder
performed to search for a mutation in the ATP7A gene. There is no cure for Menkes disease. Early treatment with injections of copper supplements (acetate
Menkes_disease
Spectrum of conditions caused by HIV infection
is a preventable disease. It can be managed with treatment and become a manageable chronic health condition. While there is no cure or vaccine for HIV
HIV/AIDS
Surgery used to treat skin cancers
removal of skin cancer with a very narrow surgical margin and a high cure rate. The cure rate with Mohs surgery cited by most studies is between 97% and 99
Mohs_surgery
Plant disease of bananas
Panama disease (or Fusarium wilt) is a plant disease that infects banana plants (Musa spp.). It is a wilting disease caused by the fungus Fusarium oxysporum
Panama_disease
Disease involving the vertebral column
Spinal disease refers to a condition impairing the backbone. These include various diseases of the back or spine ("dorso-"), such as kyphosis. Dorsalgia
Spinal_disease
Disorders of adaptive immune system
of the condition. The majority of the autoimmune diseases are chronic and there is no definitive cure, but symptoms can be alleviated and controlled with
Autoimmune_disease
Medical condition
in the MTOR gene. Frequency of this disease is unknown, but all ethnic groups are equally affected. There is no cure for SKS, but management of some symptoms
Smith–Kingsmore_syndrome
Medical condition
is rare and unfamiliar to most physicians. It can be mistaken for other forms of heart disease and/or muscular dystrophies, including Pompe disease. Females
Danon_disease
Mitochondrial disorder
myoclonic epilepsy with ragged red fibers) is a mitochondrial disease. It is extremely rare, and has varying degrees of expressivity owing to heteroplasmy
MERRF_syndrome
Rare genetic disorder
Multiple epiphyseal dysplasia (MED), also known as Fairbank's disease, is a rare genetic disorder (dominant form: 1 in 10,000 births) that affects the
Multiple_epiphyseal_dysplasia
CURE RARE-DISEASE
CURE RARE-DISEASE
CURE RARE-DISEASE
CURE RARE-DISEASE
CURE RARE-DISEASE
CURE RARE-DISEASE
CURE RARE-DISEASE
CURE RARE-DISEASE
CURE RARE-DISEASE