According to a story from BioSpace, the biopharmaceutical company Merck recently announced that the US Food and Drug Administration (FDA) has approved the company's cancer drug KEYTRUDA in a new…
According to a story from WebMD, a recent study revealed that men with Crohn's disease, ulcerative colitis, or other forms of inflammatory bowel disease are at an increased risk of…
According to a story from BioSpace, a report on the results of the Orphan Drug Act of 1983 that was originally commissioned by the National Organization for Rare Disorders (NORD)…
According to a story from CBS News, Roxli Doss, an eleven year old girl, was diagnosed with a rare and dangerous brain tumor called diffuse intrinsic pontine glioma. It is…
Patients, caregivers, and advocates alike all know how devastating and exhausting a rare disease can be, so it's important to find the moments to celebrate when someone overcomes the odds…
This is an interview I did with Scott Davidson who has a podcast called Living Adaptive, where he interviews people that have adapted to significantly difficult situations in their lives.…
According to a story from Nature, Dr. Elliot Vichinsky thinks that almost a third of his patients with sickle cell disease ultimately die from causes that could be prevented. Sickle…
When my family friend's new girlfriend came to meet us, she made the best impression with her positive attitude and general ease in interacting with a large (and probably overwhelming)…
Immune Thrombocytopenia (ITP) is diagnosed by low platelet counts in the blood. It also causes impaired platelet production. This put patients at risk for serious bleeding as well as spontaneous bruising.…
According to a story from Market Screener, the drug developer Albireo Pharma, Inc., recently announced that its investigational product candidate A4250 has earned Orphan Drug designation from the European Commission.…
According to a story from the Translation Genomics Research Institute (TGen), the organization was recently awarded a grant of $10,000 from PayPal Gives. The funding will be allocated towards funding…
According to a story from PR Newswire, the genomics medical company Sangamo Therapeutics, Inc., recently announced that the first patient had been dosed in its Phase 1/2 clinical trial which…
According to a story from EurekAlert!, monitoring lung function is an important role of managing Duchenne muscular dystrophy and other diseases that can cause wasting of the respiratory muscles. Many…
According to a scientific study published in the Orphanet Journal of Rare Diseases, a team of researchers were able to identify the mutations responsible for choroideremia in five families of Polish descent.…
According to a story from freepressseries.co.uk, 45-year-old Stephen Millichamp was recently diagnosed with AL amyloidosis. The news was dire, and Stephen was told that the disease would ultimately be fatal.…
Hope everyone's holiday season is off to a good start! As we gear up for Christmas and the New Year, we're bringing you four stories that stood out from this…
According to a story from HudsonAlpha Institute for Biotechnology, a team of researchers affiliated with the organization have identified a genetic mutation that is linked a rare pediatric form of…
According to a story from Financial Review, the US is not the only place in the world where lobbying is a central component of political activity. So far, lawmakers in…
According to a story from EurekAlert!, a team of researchers used a combination of microarray technology and blood tests in order to distinguish distinctive molecular signatures in a group of…
A new Phase 2 Parkinson's disease trial is now enrolling 42 patients who have lived with the condition four years or more. Ultimately, the trial aims to evaluate a new…
According to a story from pm360online.com, the pharmaceutical company Roche recently announced that its investigational therapy risdiplam has awarded PRIME designation from the European Medicines Agency (EMA). The drug has…
According to a story from the National Post, 34-year-old mother Andrea Cardin, a photographer, was excited to welcome her newborn son James into the world on November 21st of this…
According to a story from Oncology Nurse Advisor, a recent study found that patients with immune thrombocytopenia do not carry the same understanding as their doctors do when it comes…
At four years old, Norah Gratz-Lazarus of California is a sweet, bubbly girl, full of spirit. She's a big sister, and loves jokes, ice cream, and chocolate chip cookies. However, Norah…
According to a story from BioSpace, Incyte Corporation and Eli Lilly and Company recently announced that the US Food and Drug Administration (FDA) has given the drug baricitinib Fast Track…
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