According to a story from the Ottawa Citizen, Dianne Sauvé, who is 60 years old, learned six years ago that she has the rare condition acromegaly. Since she learned that…
Are you interested in learning about the latest trends and drug development in the rare disease field? Then you should check out a special Journalist Team Presentation from Global Data…
Neurofibromatosis is an extremely rare condition. Worldwide it affects just one in four thousand people. Gail Appelgren and Tim Golumbia are two of the individuals living with the illness and…
Epidiolex was the first cannabis-based drug approved in the United States. It was first recommended for FDA approval by an advisory committee in April of 2018. In June of this…
Society teaches us to make comparisons all of the time. When you chose to participate in a competitive game, and you want there to be a winner and a loser,…
According to a story from BioPortfolio, the biopharmaceutical company SpringWorks Therapeutics recently announced that the US Food and Drug Administration (FDA) has given the company's experimental product PD-0325901 Orphan Drug…
When a lot of people think of physical therapy, they think of rehabilitation to improve an injury or some type of ailment. However, the world of physiatry is much broader…
Acer Therapeutics has submitted a New Drug Application to the United States Food and Drug Administration on behalf of Edsivo™, their experimental treatment for vascular Ehlers-Danlos syndrome. To find out…
According to a story from Scleroderma News, many rare disease patients will be heading to the polls on November 6th. Many of them are as deeply concerned about the high…
According to a story from pm360online.com, the drug developer Bavarian Nordic A/S recently announced that its Phase 2 clinical trial of its experimental product BN-Brachyury has begun, with the first…
Forbes reports that Healx, a British biotech startup, is using the power of artificial intelligence to find new uses for existing drugs that treat rare diseases. "Healx combines AI technology, pharmacology…
According to a story from The Daily Texan, professor Marian Williams-Brown from the University of Texas is conducting research in an effort to develop a more effective approach for detecting…
RAREfest, an event organised by the Cambridge Rare Disease Network (CRDN), will bring together patients, scientists, researchers, clinicians, support groups, businesses, and the public, to engage and educate people about…
Goethe University in Frankfurt, Germany has been awarded a grant of more than one million euros as part of a large-scale project in Europe to standardise and make available clinical…
The Cambridge Rare Disease Network’s RAREfest, a festival focusing on rare diseases, will bring together researchers, scientists, patients, and the public. It’s taking place on the 30th November and 1st December in…
Growing up without a diagnosis is hard. In a world which frequents new scientific discoveries, you're still living as a medical mystery. When no one knows the answer, patients often…
Novartis has announced positive findings from their research into brolucizumab, an investigational drug being developed to treat neovascular age-related macular degeneration. This data comes from year two of the…
According to a story from greeleytribune.com, a couple has been working to form a nonprofit to help spread awareness about an unusual rare disease called achalasia, which can cause the…
For esophageal cancer (like many rare conditions), early diagnosis means a better prognosis. Unfortunately, until now there haven't been many ways to determine whether or not an individual may be at…
An innovative new treatment means practically nothing if we can't determine the type of patient it's going to be effective for. For an illness like acute myeloid leukemia (AML), which is…
NSCLC Non-small cell lung cancer or NSCLC is the most common kind of lung cancer diagnosis. It affects over 2 million people and of those, there's a small group who…
According to a story from epidermolysisbullosanews.com, the biotechnology company TWi Biotechnology, Inc., recently announced that is has begun enrollment for its Phase 2 trial. This trial will test the company's…
A major genetic research project called the Deciphering Developmental Disorders (DDD) study is helping families to receive diagnoses, and, in some cases, even identifying new disorders. For more information,…
The problem Synapses in the brain are the connections between neurons that allow them to communicate with one another. In a healthy brain at least, that's how chemical signals are successfully…
According to a story from mdmag.com, the publication recently conducted an interview with Dr. Eric Ruderman. Eric is a professor at Northwestern University with the Division of Rheumatology. The magazine…
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