Rare diseases UK list

Rare diseases UK list

Rare Disease Day provides the rare disease community across the world with the opportunity to increase awareness of rare diseases and highlight this public health priority to government and health departments. It is recognised in countries all around…

Rare diseases and symptoms list

Rare diseases and symptoms list

A disease is an abnormal and pathological condition that affects either a part or a whole organism. Although diseases have specific symptoms and signs for easier detectability, most diseases, and more often, rare diseases go by undetectable even for some…

Ultra rare diseases list

Ultra rare diseases list

Ensemble Therapeutics is putting its discovery engine to work on ultrarare diseases. The Cambridge, MA-based biotech will explore its rapidly growing library of 10 million macrocycles to see what it can whip up for Alexion s pipeline. The biotech has…

Rare disorders in infants

Rare disorders in infants

Bill would amend existing law amid concerns that testing for five other rare diseases might be stalled State Sen. Nellie Pou, sponsor of legislation that would require hospitals to screen newborns for Hunter syndrome, and Jeff Leider, whose two young…

Rare diseases list with Description FOP

Rare diseases list with Description FOP

People with rare diseases have many challenges in common, and I’ve posted about that in previous Februaries. THAT’S why Rare Disease Day helps - 1 in 10 or 12 people is facing difficulties, and Rare Disease Day raises awareness of the problem. This year…

List of rare Autoimmune diseases

List of rare Autoimmune diseases

The most common cause of all diseases in the world today is autoimmune diseases. In autoimmune disease our own protective forces (immune system) starts to attack your own body. The name of the disease depends upon the location of the organ under attack…

FDA rare diseases list

FDA rare diseases list

The 8-week reprieve from sequestration has benefited all federal discretionary programs. “Live to fight another day” is not to be taken for granted. Sequestration still, however, looms as an existential threat to the mission of the FDA. If it occurs ……

List of rare diseases in Human

List of rare diseases in Human

The voices of rare disease patient advocates play a unique and critical role in the modernization of the clinical trials process, which advocates see as antiquated and unable to sufficiently address their unmet medical needs. A slogging, expensive regulatory…

List of rare diseases companies

List of rare diseases companies

Genzyme, Shire and more hold global campaigns today A multitude of pharma companies, including Sanofi s rare disease division Genzyme, have leant their support to Rare Disease Day. The event, held on the last day of February, aims to help people with…

Rare disease common variants hypothesis

Rare disease common variants hypothesis

STEP 1: Assemble disease cohort Assemble a disease or clinical trial cohort of 100 – 1, cases. EXAMPLE – Consider that Disease X is caused by genetic variants in 3 different genes (Genes X1, X2, X3), wherein each patient has a mutation in only 1 of these…

Rare brain diseases list

Rare brain diseases list

Officials have not released the child’s name or age, saying that it would be in violation of federal law, but according to a message posted on the Facebook page called Prayers for Blake Driggers, the child passed away on Tuesday from the infection. Facts…

Rare diseases list in children

Rare diseases list in children

Rare Disease Partnering Summit Wednesday, April 24th 2:00PM - 5:00PM Don t miss the opportunity to hear these exciting Business Forum presentations by world leading pediatric research institutions! Join renowned investigators from five world-class pediatric…

Rare heart diseases list

Rare heart diseases list

Rheumatic heart disease Rheumatic heart disease is damage caused to the heart’s valves by rheumatic fever, which is caused by streptococcal bacteria. Valvular heart disease The heart’s valves keep blood flowing through the heart in the right direction…

List of orphan diseases

List of orphan diseases

The Food and Drug Administration has charged the Office of Orphan Products Development (OOPD) to dedicate its mission to promoting the development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions…

Rare respiratory diseases list

Rare respiratory diseases list

Individuals with weakened immune systems are particularly susceptible to rare respiratory diseases. Photo Credit Rare pulmonary diseases can be inherited or acquired by exposure to millions of microorganisms that populate the atmosphere. Bacteria, viruses…

Lists of Odd rare diseases

Lists of Odd rare diseases

Also known as multiple chemical sensitivity (MCS), Twentieth-Century Disease is described as a chronic condition characterized by adverse effects from exposure to low levels of chemicals or other substances in modern human environments. Suspected substances…

Center diagnosis rare genetic diseases

Center diagnosis rare genetic diseases

Your spouse has just been diagnosed with a rare genetic illness that you’ve never heard of. Your reaction might well be a mix of shock and panic, as your mind starts racing wildly and you feel that life as you have known it is coming to an abrupt halt…

Rare diseases Canada list

Rare diseases Canada list

Canada is the first country in the Americas to launch an international Web portal designed to improve research and treatments for rare diseases, Health Data Management reports. Portal Details The international portal– called Orphanet– is led by a consortium…

Interesting rare diseases list

Interesting rare diseases list

This disease, which according to the NIH is most often caused by a begnin tumor in the pituitary gland, results in an excess of GH, or growth hormone. This causes sufferers to grow abnormally large, not just in terms of height, weight but also organ size…

Rare disease database search

Rare disease database search

A collection of resources on topics of interest to the rare disease community, including rare disease social networks, online medical reference Web sites, rare disease events, and more. Basic Information Medical and Science Glossaries The following glossaries…

Rare fatal diseases list

Rare fatal diseases list

Fatal familial insomnia (FFI) is a type of genetic prion disease, also known as a transmissible spongiform encephalopathy (TSE). Prion diseases are progressive, neurodegenerative disorders that may affect humans and animals. The first symptoms of FFI…

List of rare diseases in India

List of rare diseases in India

Kyasanur Forest Disease Kyasanur Forest Disease (KFD), also referred to as monkey fever is an infectious bleeding disease in monkey and human caused by a highly pathogenic virus called KFD virus (KFDV). KFDV is of zoonotic origin (originating from animals)…

Orphan diseases list myelitis

Orphan diseases list myelitis

NMO Is a rare relapsing autoimmune disorder that preferentially causes inflammation in the optic nerve and spinal cord. It is characterized by longitudinally extensive myelitis (myelitis which is 3 vertebral segments in length or greater), which can leave…

Rare diseases of the eye list

Rare diseases of the eye list

Whether or not you believe the eyes are the window to the soul, there’s no doubt that peering into pupils can help doctors detect health problems. Some are serious and rare and can only be detected when your doctor shines a light into your eyes or an…