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…
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…
The Dupuytren Research Group was founded ten years ago. Our original name was Dupuytren Foundation. Our original mission was simple: to advocate for Dupuytren patients. Providing patient education, physician education, connecting patients with patients,…
My name is Casey. I’m 27 years old and have a 50% chance of becoming symptomatic for a neurodegenerative disease in my 30’s (if my onset aligns with that of…
According to a story from Markets Insider, Taiho Oncology, Inc. recently announced that the US Food and Drug Administration (FDA) has officially approved the company's cancer drug LONSURF in the…
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 Statesman Journal, nine year old Haley Pollman isn't so different from most other girls her age. She loves dresses, Barbie dolls, and animals, and still…
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…
According to a publication from ANCA Vasculitis News, a sizable number of people who do not have ANCA-associated vasculitis could falsely test positive due to the presence of the associated…
A press release put out last month by American pharmaceutical company Adverum Biotechnologies announced some of the long-term preclinical safety data collected about their experimental drug ADVM-022. The drug hopes…
A publication at Science Daily recently announced that findings from Children's Hospital of Philadelphia suggested that cysteamine bitartrate, already used for nephropathic cystinosis (a rare kidney disease), might potentially benefit patients…
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 finanznachrichten.de, the biopharmaceutical company Theranexus has recently released preliminary results from a Phase II clinical trial that is testing the company's experimental product THN102. This…
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…
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