Alan Jackson has been a big part of the country music scene for a long time, with songs like Remember When and Livin' On Love. Recently, he revealed to his…
In the past, caffeine has been somewhat of a complex or even controversial topic. While some research shares that it is bad for health, others laud caffeine’s ability to enhance…
In the United States, Orphan Drug designation is granted to drugs or biologics which show promise in treating patients with rare and life-threatening conditions. For the purpose of this article…
Science Direct recently published a Respiratory Medicine Case Report that may impact future Cystic Fibrosis (CF) treatment. The case involves adult brothers herein referred to for purposes of this…
Dave and Kendra Riley have a beautiful family: them and their daughters Eva, Kiera, and Olivia. Yet the family has faced some scary and difficult obstacles over the last…
Clinical trials have many endpoints - or milestones they are looking to achieve. These endpoints help signify the safety, efficacy, and tolerability of the treatments. In the Phase 3…
Having a rare disease can feel isolating. Many people have never even heard of your condition, much less know anything about it. This is even a problem with doctors. Despite…
Sometimes, certain drugs may be approved in different countries and not in others. However, drug approval spanning multiple countries highlights the importance of getting drugs and treatments into the hands…
I don’t know about you, but I always get incredibly happy when I see stories about a community rallying to support one of its own members. For example, those in…
It's no secret that COVID-19 can cause lasting effects on those it infects. Over the course of the pandemic, we've learned a lot more about this virus, including its connection…
In 1992, Elsa Burgos and her husband, living in Puerto Rico at the time, welcomed a new addition to their lives: their daughter Nicole. The pair were thrilled to have…
Within the United States, uveitis is considered a leading reason for preventable blindness. As the uveitis progresses, patients may also develop complications such as uveitic glaucoma. But this can…
It's no secret that, over the last 1.5-2 years, COVID-19 has played a huge role in all of our lives. The novel SARS-CoV-2 coronavirus is present in over 200…
Francis Collins, M.D. is the director of the NIH which is involved with a national study of long COVID. According to a report by Alaska Public Media, the NIH has…
Sometimes, it can be difficult to spur drug development for rare or life-threatening diseases, considering the number of patients. To address this, and improve patient outcomes, the FDA developed the…
September will soon come to a close, but did you know that this month is also Ovarian Cancer Awareness Month? According to a story from MSN, Michelle Barrenechea, an EMT…
Have you ever had a panic attack? If so, you know how terrifying they can be. I have had plenty and know exactly what they are like…torture! There is good…
On September 21, 2021, Rare Disease Legislative Advocates (RDLA) hosted its monthly webinar. The focus of this webinar was newborn screening in recognition of Newborn Screening Awareness Month, which is…
According to a story from MSN, Busola Omiyale's daughter Toriayooluwa was only nine months old when she could tell that something wasn't right. The girl had sickle cell disease, a…
Funding is a problem that many rare diseases face when it comes to research. Cholangiocarcinoma, also known as bile duct cancer, is no exception. Because of this, it is often…
Unfortunately, clinical trials do not always proceed the way that drug developers - and patients - hope. For example, an ongoing Phase 1/2 clinical trial evaluating BMN 307, an investigational…
Nucala has already been approved in Europe as an add-on treatment for patients with extreme eosinophilic asthma. Now, the EMA's Committee for Medicinal Products for Human Use (CHMP) has recommended…
According to a recent news item that appeared on The Denver Channel, we are now in the age of genetic research that could have an impact on the lives of…
Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest…
Jeff was first diagnosed with systemic mastocytosis early in life, at age 16. The first sign that Jeff knew that something was off was when he developed urticaria pigmentosa, abnormal…
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