Wisconsin; the land of beer, cheese, good-hearted people, and Maria Voermans - rare cancer survivor and now fierce advocate for the rare disease community. Ten years ago, Maria had the…
Genetic testing, such as 23andMe, has been approved by the FDA for direct-to-consumer genetic testing. However, this type of testing has not been approved to predict rheumatic diseases. Research is…
Thanks to a grant from Worldwide Cancer Research, as well as support from various other organizations, researchers at UCLA recently uncovered a new, faster, easier, and more personalized way to…
Most biological drugs, including insulin, can't be taken orally, because enzymes and proteins cause the drugs to break down too quickly in the digestive system. For decades, the pharma industry…
Although each disease on its own may be rare, a huge number of people are living with a rare disease-- and half of these people are children. About 30 percent of these children…
According to a story from The Indian Express, doctors and drug developers are at a crossroads over an innovative form of therapy called fecal microbiota transplants. These transplants are essentially…
According to a story from Xtalks, there has been an uptick in research related to rare diseases and the development of new therapies for them. Despite this, only a small…
Problems in China Around the world there are 7,000 rare diseases currently identified. Of these, researchers have uncovered effective treatment options for less than 10%. Sadly, even when diseases have…
The State of Rare Disease in India There are 7,000 known rare diseases worldwide. The Organization for Rare Diseases India (ORDI) estimates 70 million people are affected by a rare diagnosis…
SOJIA SOJIA stands for systemic-onset Juvenile Idiopathic Arthritis (JIV). It is a very severe form of JIA which causes joint pain, inflammation, recurrent fevers, and a skin rash. Patients often take predinsolone,…
There are over 500 children in Scotland whose parents feel could benefit from a new cannabis-derived drug for seizures called Epidiolex. But recently, Secretary Jeanne Freeman had to tell…
According to a story from news-medical.net, a team of scientists associated with the CURECN project are starting to develop a gene therapy that has the potential to cure the rare…
On February 24, 2019, the Organization for Rare Diseases India, USA (ORDIUSA) held a 7km walk/run at four different locations across the United States. The Event was called Racefor7 and its goal…
Each year, Rare Disease Day (February 28th) incites individuals and organizations to investigate and reform their rare disease efforts. This year, the EU Commission has made drastic changes which should…
According to a story from Ottawa Citizen, the CHEO Research Institute has recently begun operations at its Centre for Genomic Innovation. Much of the the center's operations will focus on…
On December 28, 2018, five-year-old Oscar Saxelby-Lee was diagnosed with a rare and aggressive form of leukemia. Now he's in a race against time to find an eligible stem cell…
Happy Thursday! Today, we're highlighting an article on a story about rare disease discrimination, followed by news about a musician who went public with a rare diagnosis. After that, we…
"...just like 50,000 other people in the United States each year, this week I was diagnosed with stage 4 pancreatic cancer." Alex Trebek - who has has hosted the…
According to a story from EurekAlert!, a team of scientists has discovered a genetic variation that can cause amyotrophic lateral sclerosis. This variant acts on a biologic pathway that has…
Familial Chylomicronemia Syndrome Familial chylomicronemia syndrome (FCS) is a condition that is considered ultra-rare. It's caused when the enzyme called lipoprotein lipase (LPL) is impaired. It is can result in…
According to a story from Gaucher Disease News, a recently published case report study suggests that instances of splenic artery aneurysm are being underreported in patients with Gaucher disease. The…
A Collaborative Effort An international team of researchers has just uncovered the genetic cause for an ultra-rare form of leukoencephalopathy. Leukoencephalopathy refers to a family of conditions which affect the…
About GOSH The Great Ormond Street Hospital (GOSH) is both a pediatric hospital, treating many children with rare conditions, and a charity organization for patients like those they care for.…
The European Medicines Agency (EMA) recently published a press release in which the agency's human medicines committee, known as CHMP, as recommended the authorization of a new treatment for a…
According to a story from Central Charts, the biopharmaceutical company Acceleron Pharma Inc. recently announced that the company's experimental drug candidate ACE-083 has earned Orphan Drug designation from the US…
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