Cure Rare Disease information

Cure Rare Disease is a non-profit biotechnology company based in Boston, Massachusetts that is working to create novel therapeutics using gene therapy...

A rare disease is a disease that affects a small percentage of the population. In some parts of the world, the term orphan disease describes a rare disease...

Platform for Rare Disease Registries (EPIRARE) The World Association of Orphan Diseases (WAO(R)D) The World Association of Cured Rare Diseases (WACRD) Care-For-Rare...

Retrieved 2023-08-25. "Cure Rare Disease Collaboration with Taconic Biosciences Produces Novel ADSSL1 Mouse Model | | Cure Rare Disease". www.cureraredisease...

phenomenon. Incurable disease A disease that cannot be cured. Incurable diseases are not necessarily terminal diseases, and sometimes a disease's symptoms can...

also called Münchmeyer disease or formerly myositis ossificans progressiva, is an extremely rare connective tissue disease in which fibrous connective...

ASIN B000J0QZSC "Les malades imaginés: Diseases in fiction". René Krémer. Acta Cardiologica, 2003. No Cure for the Future: Disease and Medicine in Science Fiction...

Lafora disease is a rare, adult-onset and autosomal recessive genetic disorder which results in myoclonus epilepsy and usually results in death several...

as mucopolysaccharidosis type III (MPS III), is a rare autosomal recessive lysosomal storage disease that primarily affects the brain and spinal cord....

Fatal insomnia is an extremely rare neurodegenerative prion disease that results in trouble sleeping as its hallmark symptom. The majority of cases are...

sclerosis (ALS), also known as motor neurone disease (MND) or Lou Gehrig's disease in the United States, is a rare but terminal neurodegenerative disorder...

Canavan disease, or Canavan–Van Bogaert–Bertrand disease, is a rare and fatal autosomal recessive degenerative disease that causes progressive damage to...

(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...

fatal. 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...

prevent misdiagnosis and inappropriate treatment. Kikuchi's disease is a very rare disease mainly seen in Japan. Isolated cases are reported in North America...

FTD-MND). Two distinct rare subtypes are neuronal intermediate filament inclusion disease (NIFID), and basophilic inclusion body disease (BIBD). Related disorders...

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...

Subacute sclerosing panencephalitis (SSPE), also known as Dawson disease, is a rare form of progressive brain inflammation caused by a persistent infection...

Castleman disease (CD) describes a group of rare lymphoproliferative disorders that involve enlarged lymph nodes, and a broad range of inflammatory symptoms...

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...

include funding research on treatment and cures for rare diseases; lobbying for legislation to benefit the rare diseases community (in addition to the Orphan...

became rare following occupational exposure limits established around 1950. Berylliosis is an occupational lung disease. While there is no cure, symptoms...