Although there is no cure for this disorder, there are treatments that can help alleviate symptoms.
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Articles on Rare diseases
Displaying 1 - 20 of 21 articles
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.
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.
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 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.
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.
Despite the fact that rare diseases aren’t actually so rare, it appears they suffer from a branding problem in Canada.
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.
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.
Rare diseases may only affect a handful of people but their treatment benefits everyone.
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.
Policy moves to benefit patients are now benefiting business.
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.
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.
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…
Top contributors
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Senior Research Fellow, Neuroscience Research Australia
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John Curtin Distinguished Professor, Molecular and Life Sciences, Curtin University
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Curtin Research Fellow, Curtin University
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Professor of Veterinary Infectious Diseases, University of Liverpool and International Livestock Research Institute, Kenya, University of Liverpool
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International adviser and Fellow, Royal College of Pathologists
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Researcher and Head of Division in Earth Sciences, Natural History Museum
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Professor and Director of the Clinical Anatomy Learning Centre, Lancaster University
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Research Fellow, Cardiff University
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Professor, The University of Queensland
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Professor of Pharmacoeconomics, Co-director of the Centre for Health Economics & Medicines Evaluation, Bangor University
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Senior Lecturer in Accounting and Finance, University of Liverpool
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Professor, Children's Cancer Institute
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Associate Professor (conjoint), Children's Cancer Institute
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Professor Emeritus of Health Policy and Management, York University, Canada
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Professor of Pharmacy Practice, University of Connecticut
