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…
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…
According to a story from The Muscular Dystrophy Association (MDA), the organization alongside the Charot-Marie-Tooth Association (CMTA) has awarded grant funding totaling $276,430 to a research study that will develop…
According to a story from news-medical.net, the University Hospitals in Cleveland, Ohio and the University of Oxford have recently announced their plans for a multi-year affiliation. The goal of this…
According to a story from The Irish Times, a recent report issued by the Irish Pharmaceutical Healthcare Association (IPHA) indicates that Ireland trails many other European countries in approving new…
Not only are rare diseases well, rare, but the same rare disease can also affect different patients in a multitude of ways. For instance, some diseases can impact many different…
According to a story from news-medical.net, a research team affiliated with Princeton University has been hard at work developing a unique tool that utilizes machine learning in order to help…
According to a story from ASH Clinical News, the treatment of younger patients with multiple myeloma, a form of rare blood cancer, requires physicians to take into account certain factors…
Jordan Flake and her son Jackson were on their way from El Paso, Texas to Dallas, Texas when they were kicked off their flight because of their Ichthyosis diagnosis. They were on…
Ipsen has just announced that they will be acquiring Clementia Pharmaceuticals. This merging of companies is exciting for the rare community because it will strengthen the resources, knowledge-base, and overall…
According to a story from Business Insider, a recent study has found that a team of doctors has a much better chance of making an accurate diagnosis than a single…
According to a story from The Indian Express, a young baby at the Sir Ganga Ram Hospital in Delhi was just diagnosed with an incredibly rare disease that has less…
According to a story from Kractivist, 38 year old Payel Bhattacharya has the rare genetic disorder von Hippel-Lindau disease, or VHL. Payel has written a book about her life called A…
According to a story from Regulatory Affairs Professionals Society, a recent study found that the research and development costs for new molecular entities that were classified as orphan drugs was…
According to a story from BioPortfolio, the biotechnology company Saniona has released an update in regards to the company's Phase 2a clinical trial of Tesomet, which is being tested as…
Rare disease isn't as rare as it sounds. Ten percent of Americans have a rare disease-- and there are almost 7,000 known rare diseases. A rare disease, also known as…
According to a story from Fierce Biotech, the results of a year-long study examining the utility of the groundbreaking gene-editing technology CRISPR in treating the rare genetic disorder Duchenne muscular…
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