Celine Dion, performing in London on July 5, 2019. Since her diagnosis, she has rescheduled her tour dates.
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Although there is no cure for this disorder, there are treatments that can help alleviate symptoms.
The blood vessel dilation caused by sildenafil (Viagra) can be beneficial in lung diseases such as pulmonary arterial hypertension or idiopathic pulmonary fibrosis.
Sildenafil — better known as Viagra — may be helpful in treating lung diseases like pulmonary arterial hypertension and idiopathic pulmonary fibrosis, for which there are few effective treatments.
The National Institutes of Health estimates the existence of 7,000 rare diseases, with some affecting only a handful of people.
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Deciphering the biological pathways behind rare genetic diseases often involves assembling a team of specialists to work closely with the family members of those affected.
About 1 in 300 people in the general population carry the Tay-Sachs disease gene.
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Tay-Sachs is a rare and fatal neurodegerative disorder that most commonly affects children. Researchers have developed the first Tay-Sachs treatment to reach clinical trials.
Spinal muscula atrophy causes muscle wasting and loss of control, usually starting in infancy.
Spinal muscular dystrophy affects at least 1 in 10,000 people, but new drugs have given hope to those suffering from this rare disease.
A rare disease is one that affects less that five in 10,000 people. There are about 7,000 known rare diseases.
Canadian finance minister Bill Morneau announced funding for a new Canadian Drug Agency in the 2019 Federal Budget. Here he speaks at a press conference in Toronto, March 20, 2019.
THE CANADIAN PRESS/Cole Burston
A new agency and money for drugs for rare diseases are only very partial steps on the road towards what Canada really needs: a national pharmacare plan.
Rare diseases aren’t, in fact, all that rare. Yet they continue to be brushed aside by most politicians. Why?
Despite the fact that rare diseases aren’t actually so rare, it appears they suffer from a branding problem in Canada.
In 2030, some diseases are defined more specifically than in the past with a focus on their molecular makeup. This is known as precision medicine.
In 2030, there is a boom in precision medicine, where diseases – from cancer to dementia – are defined and targeted more specifically with a focus on their molecular makeup.
If your baby has a rare disease, should we put a limit on how much to spend on the drug that may save her?
Specialty prescription drugs are responsible for countless medical miracles, but their high price tag is the main reason health care costs are out of control.
DNA holds the secrets of human existence, and studying rare diseases can reveal some of these facts of life.
Rare diseases may only affect a handful of people but their treatment benefits everyone.
Childhood cancer desperately needs more research.
Children’s cancer is a rare disease, which means the market is small and pharmaceutical companies have few incentives to develop drugs for these cancers.
Rare diseases have turned into big business.
Policy moves to benefit patients are now benefiting business.
Robert Wadlow is known as the tallest man who ever lived.
Gigantism and acromegaly are rare conditions where a benign tumour causes excessive production of growth hormone (GH), which stimulates the growth of the skeleton and all tissues in the body.
Rare diseases are yielding secrets about very common conditions.
By definition, a rare disease isn’t always easy to spot but there are ways to make it easier to do.
A typical elephant shark from the Melbourne Aquarium.
Some things that develop as normal in elephant sharks and other marine life can mimic things we see in human disease. That makes these ‘mutants’ ideal for study to find out why things go wrong in humans.
African camels could prove to be an important source of information about the MERS virus.
There is a high prevalence of the MERS virus in African camels. More research is key to understanding the virus better globally.
Researchers have found that mexiletine, an anti-arrhythmic medication now rarely prescribed, is effective at easing pain…
Patients with the rare genetic disorder X-linked lymphoproliferative disease (XLP) are in some cases living longer thanks…