Warmer ocean waters are fueling the spread of the bacteria Vibrio vulnificus. Infections can lead to a rare but fatal condition called necrotizing fasciitis.
Articles on Rare diseases
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Scotland is now screening every newborn for a condition that can kill within two years. Here is what parents across the UK need to know.
A small but growing number of Australians are being diagnosed with a rare tattoo-related eye condition.
Seles’ first symptoms appeared suddenly around three years ago. She began having double vision and weakness in her arms and legs.
The incurable autoimmune condition affects an estimated 3-12 in every 100,000 people.
Great Ormond Street Hospital plans to apply to the UK drugs regulator for a gene therapy to cure a rare genetic disorder called ADA-SCID.
Drug development takes a great deal of time, money and effort. While future profits play a big factor in which diseases gets prioritized, advocacy and research incentives can also tilt the scale.
Warmer ocean waters are fueling the spread of the bacteria Vibrio vulnificus. Infections can lead to a rare but fatal condition called necrotizing fasciitis.
Animals, including the ones that live in our homes, can carry all kinds of illnesses. Most of the time it’s not a problem, but here’s what you should do to avoid getting sick.
Spinal muscular atrophy, a disease the author of the article suffers, is one of the genetic diseases with the highest associated mortality. It is also the most expensive to cure.
Although there is no cure for this disorder, there are treatments that can help alleviate symptoms.
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.
Record-breaking technology can sequence an entire human genome in a matter of hours. The work could be a lifeline for people suffering from the more than 5,000 known rare genetic diseases.
Spinal muscular dystrophy affects at least 1 in 10,000 people, but new drugs have given hope to those suffering from this rare disease.
‘Orphan drugs’ with high price points are being tested as treatments for COVID-19. There’s a better way to spur low-cost innovation for new drugs.
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.
Top contributors
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Distinguished Professor of Pharmacy Practice, University of Connecticut
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Professor of Anatomy, Lancaster University
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Professor of Microbiology and Immunology, Indiana University
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Research Fellow, Cardiff University
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Professor of Pharmacoeconomics, Co-director of the Centre for Health Economics & Medicines Evaluation, Bangor University
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Associate Professor (conjoint), Children's Cancer Institute
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John Curtin Distinguished Professor, Molecular and Life Sciences, Curtin University
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Professor, Central Clinical School, Monash University
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Professor, The University of Queensland
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Researcher and Head of Division in Earth Sciences, Natural History Museum
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Senior Lecturer in Accounting and Finance, University of Liverpool
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Senior Research Fellow, Neuroscience Research Australia
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Professor, Children's Cancer Institute
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Associate Professor of Neurology, UMass Chan Medical School
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Curtin Research Fellow, Curtin University